| Catalyst | Drug/Treatment | Stage | Probability of Approval | Description | Drug Type | Therapeutic Area | Source |
|---|---|---|---|---|---|---|---|
PDUFA Date example | PDUFA | — | Subscribe to access the data. | Gene Therapies | Rare Diseases | ||
PDUFA Date example | PDUFA | — | Subscribe to access the data. | Gene Therapies | Rare Diseases | ||
PDUFA Date example | PDUFA | — | Subscribe to access the data. | Gene Therapies | Rare Diseases |
Recent Updates
Recently added Catalysts
Ultragenyx Pharmaceutical Inc.$33.82
-0.79 (-2.28%)
C 50Pipeline Score Fair Value Pharma · Commercial
Market Cap
2.30 B
EPS
-6.11
P/E Ratio
-
Value Trade
40.12 M
SEC Financials
Q1 2026Dilution Risk
Revenue
136.00 M
R&D Expenses
187.00 M
Operating CF
-197.00 M
Total Assets
1.30 B
Total Liabilities
1.52 B
Equity
-236.00 M
D/E Ratio
12,345
-9.02 %
Week
-3.78 %
1 Month
8.49 %
3 Month
-25.79 %
6 Month
-79.38 %
5 Year
-48.93 %
All Time
Cash Data
Cash Position
413.00 M
Monthly Burn
65.67 M
Runway
4.4 mo
Burn Trend
SEC Filing
May 6, 2026
Overview
Volume
1.58 M
52 Week Range
18.29 - 42.37
% held by Insiders
8.95 %
% held by Institutions
95.68 %
Enterprise Value
3.09 B
Total Shares
98.46 M
Short %
17.36 %
Float Shares
93.44 M
Company Description
locked
Upcoming Catalyst
Catalyst Timeline
Dated clinical, regulatory & corporate events for Ultragenyx Pharmaceutical Inc.
811Total events
2Upcoming
243Tier-1 (high impact)
2012 – 2028Coverage
Upcoming catalysts 2
Event history 809
Jun 9, 2026
◆Oral PresentationPresentation
Ultragenyx to participate in fireside chat at Goldman Sachs 47th Annual Global Healthcare Conference
May 5, 2026
Apr 6, 2026
◆Quarterly UpdateCorporate
Class action lawsuit filed; lead plaintiff application deadline April 6, 2026
Apr 2026
▲FDA Filing AcceptedUX111Regulatory Filing
FDA accepted resubmitted BLA for UX111
Sanfilippo syndrome type Asource ↗
Mar 12, 2026
▲Topline ReadoutDTX301Clinical Data
Positive 36-week topline data from Phase 3 study of DTX301
Ornithine Transcarbamylase (OTC) Deficiencysource ↗
Mar 12, 2026
▲Primary Endpoint MetDTX301Clinical Data
Primary endpoint of ammonia control met at Week 36
Ornithine Transcarbamylase (OTC) Deficiencysource ↗
Mar 12, 2026
▲Full ResultsDTX301Clinical Data
Full 36-week results including secondary endpoints and safety
Ornithine Transcarbamylase (OTC) Deficiencysource ↗
Drug Pipeline Intelligence
C50
Pipeline Score $1.2B
Pipeline ValueFair Value
Valuation Signal17
Drugs Scored1.7x
rNPV / MCap Top 75%
Small Cap (rank 229 of 911)
Percentile RankUltragenyx Pharmaceutical Inc. carries a moderate pipeline score (50/100), with $3.9B risk-adjusted pipeline value, led by Triheptanoin in Adult Polyglucosan Body Disease (Phase 2), but cash runway is a concern.
Showing 1 of 1 assets
| Drug | Indication | Phase | NCT ID | PTRS | rNPV | Status | Enrollment | Velocity | Design | Completion | ML Signal | Last Change |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Triheptanoin Small molecule | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Phase 3 | NCT05933200 | 50% | $1.5B | ACTIVE NOT_RECRUITING | 69 | STALLED | B (65) | Aug 1, 2027 | MODERATE_RISK | LOW May 26, 2026 |
Clinical Trial Results
| Drug Name | Indications | Phase | Date | Trial Results Summary | Title | Source |
|---|---|---|---|---|---|---|
— | Osteogenesis Imperfecta | Phase 3 | 2026-03-30 | statistically significant reduction in annualized fracture rate Read More | RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law Firm Read More | |
— | Osteogenesis Imperfecta | Phase 3 | 2026-03-30 | statistically significant reduction in annualized fracture rate Read More | RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law Firm Read More | |
— | Osteogenesis Imperfecta | Phase 3 | 2026-03-30 | statistically significant reduction in annualized fracture rate Read More | RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law Firm Read More |
Inside Trades
YEARLY INSIDER TRANSACTIONS
Sector Avg.
INSIDERS
SOLDINSIDERS
BOUGHT
SOLDINSIDERS
BOUGHT
POSITIVE SENTIMENT Based on 22 Insiders Transactions
Hedge Funds
Shares Held
HEDGE FUNDS
SOLDHEDGE FUNDS
BOUGHT
SOLDHEDGE FUNDS
BOUGHT
POSITIVE SENTIMENT Based on 27 hedge funds in the last quarter
18 buying (3 new)·9 selling (1 exited)·2 unchanged
Hedge Funds invested in RARE
| HedgeFund Name ( 3 ) | % of Portfolio | Current MV - | Shares Owned - | Activity Avg Price $0 |
|---|---|---|---|---|
Example Capital Management | 2.5 % | 15.00 M | 250.00 K | |
Example Capital Management | 2.5 % | 15.00 M | 250.00 K | |
Example Capital Management | 2.5 % | 15.00 M | 250.00 K |
RARE Institutional Ownership Trends
Current Insider %
5.20%
—+0.00%
Current Institutional %
62.40%
—+0.00%
Total Ownership
67.60%
Insider + Institutional
Data Points
1
1 Ticker(s)
Option Chain Statistics
| Expiration | Volume | Open Interest | Implied Volatility Calls | Implied Volatility Puts | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Calls | Puts | Put-Call Ratio | Calls | Puts | Put-Call Ratio | IV | OiWaIv | VWaIv | IV | OiWaIv | VWaIv | |
| 2026-07-17 | 0 | 0.00 | 0 | 0 | - | - | - | - | - | - | ||
| 2026-07-17 | 0 | 0.00 | 0 | 0 | - | - | - | - | - | - | ||
| 2026-07-17 | 0 | 0.00 | 0 | 0 | - | - | - | - | - | - | ||
Option Chain
| Calls | Strike | Puts | ||||
|---|---|---|---|---|---|---|
| Last Price | Volume | Open Interest | Last Price | Volume | Open Interest | |
| No data available | ||||||
Open interest
Today's Open Interest
1.00 M
Put-Call Ratio
1.1
Put Open Interest
480.00 K
Call Open Interest
520.00 K
Open Interest Avg (30-day)
900,000
Today vs Open Interest Avg (30-day)
11.11%
Option Volume
Today's Volume
750.00 K
Put-Call Ratio
0.95
Put Volume
360.00 K
Call Volume
390.00 K
Volume Avg (30-day)
800,000
Today vs Volume Avg (30-day)
-6.25%
Company News
RARE▼ -2.3%today
Ultragenyx Releases 2025 Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact
Ultragenyx Pharmaceutical Inc. released its 2025 Impact Report, highlighting its commitment to rare disease patients and innovation. The report details advancements in five investigational therapies and ongoing support for patients lacking access to approved treatments. Key achievements include significant investments in R&D and participation in global advocacy events, reflecting the company's dedication to improving patient outcomes.
Read more →RARE▲ +5.8%on this news
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Read more →RAREUltragenyx to Participate at Goldman Sachs 47th Annual Global Healthcare Conference
Ultragenyx Pharmaceutical Inc. will participate in the Goldman Sachs 47th Annual Global Healthcare Conference on June 9, 2026. CEO Emil Kakkis will engage in a fireside chat, which will be available via live and archived webcast. The company focuses on developing therapies for rare genetic diseases and has a strong commitment to efficient drug development.
Read more →RAREUltragenyx Reports First Quarter 2026 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. reported a total revenue of $136 million for Q1 2026, with significant contributions from Crysvita and Dojolvi. The company reaffirmed its financial guidance for the year, aiming for total revenue between $730 million and $760 million. Promising long-term data for GTX-102 in Angelman syndrome was highlighted, with Phase 3 results expected later this year. However, the company also reported a net loss of $185 million, reflecting increased operating expenses.
Read more →RAREUltragenyx to Participate at Bank of America’s 2026 Healthcare Conference
Ultragenyx Pharmaceutical Inc. will participate in Bank of America's 2026 Healthcare Conference on May 12, 2026. CFO Howard Horn and Chief of Staff Joshua Higa will lead a fireside chat, which will be accessible via webcast. The company focuses on developing therapies for rare genetic diseases and aims to address high unmet medical needs.
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com Ultragenyx Reports First Quarter 2026 Financial Results and Corporate Update First quarter total re
Ultragenyx Pharmaceutical Inc. reported first-quarter 2026 revenues of $136 million, reaffirming its financial guidance for the year. The company noted robust performance in Crysvita and Dojolvi sales. With promising long-term data from the GTX-102 study for Angelman syndrome, the firm is preparing for a pivotal Phase 3 readout later this year. However, the company faces financial challenges with a net loss of $185 million for the quarter, emphasizing ongoing investment needs despite growth prospects.
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2026 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will host a conference call on May 5, 2026, to discuss its financial results for Q1 2026. The call will provide insights into the company's performance and updates on its corporate strategy. Interested parties can access the live and recorded webcast through the company's website.
Read more →RAREUltragenyx Pharmaceutical Inc. Class Action: Levi & Korsinsky Reminds Ultragenyx Investors of the Pending Class Action Lawsuit with a Lead Plaintiff Deadline of April 6, 2026 – RARE
Levi & Korsinsky, LLP has announced a class action lawsuit against Ultragenyx Pharmaceutical Inc. for securities fraud, focusing on the company's misleading statements regarding the efficacy of its drug setrusumab. The lawsuit seeks to recover losses incurred by investors between August 3, 2023, and December 26, 2025. Following the announcement that the Phase III studies did not meet primary endpoints, Ultragenyx's stock experienced a dramatic decline of 42.32% in one day. Investors have until April 6, 2026, to file for lead plaintiff status in the class action.
Read more →RAREULTRAGENYX URGENT DEADLINE: Bragar Eagel & Squire, P.C. Reminds Ultragenyx (NASDAQ:RARE) Investors of the April 6th Lead Plaintiff Deadline and Urges Investors to Contact the Firm
Bragar Eagel & Squire, P.C. announced a class action lawsuit against Ultragenyx Pharmaceutical Inc. for allegedly misleading investors about the efficacy of setrusumab in patients with Osteogenesis Imperfecta. The lawsuit claims that Ultragenyx made positive statements regarding the Phase III Orbit and Cosmic studies while concealing material facts that affected the studies' outcomes. Following a disappointing announcement about the studies' failure to achieve statistical significance, Ultragenyx's stock saw a substantial drop. Investors have until April 6, 2026, to apply to become lead plaintiffs in the case.
Read more →RAREULTRAGENYX DEADLINE: ROSEN, RECOGNIZED INVESTOR COUNSEL, Encourages Ultragenyx Pharmaceutical Inc. Investors to Secure Counsel Before Important April 6 Deadline in Securities Class Action – RARE
Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit triggered by claims of misleading statements regarding the Phase III results for setrusumab, which is being studied for Osteogenesis Imperfecta. The lawsuit asserts that while the company conveyed confidence in the drug's efficacy, it concealed significant risks that could undermine those claims. Investors during the specified class period are being urged to secure legal counsel before the April 6 deadline to potentially recover losses incurred from buying shares at inflated prices. The allegations highlight a significant divergence between the company's public statements and the actual efficacy data from the ongoing studies.
Read more →RAREUltragenyx Shareholder Alert: ClaimsFiler Reminds Investors With Losses In Excess Of $100,000 Of Lead Plaintiff Deadline In Class Action Lawsuit Against Ultragenyx Pharmaceutical Inc. - RARE
Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit due to alleged failures in disclosing material information during the period of August 3, 2023, to December 26, 2025. This comes in the wake of disappointing results from its Phase 3 studies of setrusumab for osteogenesis imperfecta, which did not meet expected outcomes, leading to a 42% drop in stock price. Investors with losses over $100,000 are urged to file lead plaintiff applications by April 6, 2026. The lawsuit is being heard in the Southern District of New York.
Read more →RAREUltragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RAREULTRAGENYX PHARMACEUTICAL INC. (NASDAQ: RARE) CLASS ACTION DEADLINE APPROACHING: Berger Montague Advises Investors to Inquire About a Securities Fraud Class Action by April 6, 2026
Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit related to securities fraud for allegedly misleading investors about the results of clinical studies for its treatment of Osteogenesis Imperfecta. The lawsuit claims that the company raised expectations about the effectiveness of the ORBIT and COSMIC studies, which did not significantly reduce clinical fracture rates. Following this revelation, shares of Ultragenyx fell sharply, losing over 42% of their value. Investors who purchased shares during the specified Class Period are encouraged to seek representation by April 6, 2026.
Read more →RAREUltragenyx Pharmaceutical Inc. Notice of April 6, 2026 Application Deadline for Class Action Lawsuit - Contact Lewis Kahn, Esq. at Kahn Swick & Foti, LLC, Before Application Deadline
Kahn Swick & Foti, LLC has filed a class action lawsuit against Ultragenyx Pharmaceutical Inc., alleging securities fraud affecting investors between August 3, 2023, and December 26, 2025. This follows the company's announcement that its Phase III Orbit and Cosmic studies for the drug setrusumab failed to show meaningful efficacy in treating osteogenesis imperfecta, leading to a significant stock price drop of approximately 42%. Investors are urged to contact the law firm before the April 6, 2026 application deadline to be considered for lead plaintiff status.
Read more →RAREUltragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy
Ultragenyx Pharmaceutical Inc. has received FDA clearance for its IND application for UX016, a prodrug designed to treat GNE myopathy. This investigational therapy aims to improve sialic acid delivery to muscle, addressing a critical need in a patient population with no approved treatments. A Phase 1/2 clinical study is set to begin in late 2026.
Read more →RARERARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law Firm
The Schall Law Firm has initiated a class action lawsuit against Ultragenyx Pharmaceutical Inc. for alleged securities fraud. This lawsuit pertains to misleading statements made by the company regarding its drug candidate for Osteogenesis Imperfecta. The claims arose after the Phase III ORBIT study did not yield statistically significant results, leading to investor losses during the class period from August 2023 to December 2025.
Read more →RAREULTRAGENYX DEADLINE: ROSEN, TOP-RANKED INVESTOR COUNSEL, Encourages Ultragenyx Pharmaceutical Inc. Investors with Losses in Excess of $100K to Secure Counsel Before Important April 6 Deadline in Securities Class Action - RARE
Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit filed by investors who purchased stock between August 3, 2023, and December 26, 2025. The lawsuit alleges that the company made materially false and misleading statements about its treatment, setrusumab, particularly concerning its efficacy in reducing the annualized fracture rates in Osteogenesis Imperfecta patients. Investors are encouraged to seek qualified legal counsel before the April 6, 2026, deadline to join the class action. The Rosen Law Firm highlights the importance of selecting experienced counsel when participating in such cases.
Read more →RAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) - March 20, 2026
Ultragenyx Pharmaceutical Inc. has granted 10,839 restricted stock units to two new non-executive officers as part of its Employment Inducement Plan. This grant, approved by the compensation committee, aims to attract talent in line with Nasdaq Listing Rule 5635(c)(4). The stock units will vest over four years, contingent on continued employment.
Read more →RAREUltragenyx Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc. announced positive results from its Phase 3 study of DTX301, an AAV8 gene therapy for OTC deficiency. At 36 weeks, DTX301-treated patients showed a significant 18% reduction in plasma ammonia levels compared to placebo. The therapy was well tolerated, with manageable side effects, and improvements in overall symptoms were noted. Further data is expected in 2027.
Read more →RARE▲ +6.4%on this newsshared move
Ultragenyx Pharmaceutical Inc. (RARE) Investors: April 6, 2026, Filing Deadline in Securities Fraud Class Action - Contact Kessler Topaz Meltzer & Check, LLP
Ultragenyx Pharmaceutical Inc. (RARE) is facing a securities fraud class action lawsuit filed from August 3, 2023, to December 26, 2025. The lawsuit alleges that the company made misleading statements about its drug setrusumab and failed to disclose key risks from its Phase III studies. Affected investors have until April 6, 2026, to seek lead plaintiff status. The company's stock has already suffered a significant decline due to disappointing study results revealed on December 29, 2025.
Read more →RARE▲ +6.4%on this newsshared move
Ultragenyx Pharmaceutical Inc. Investors with Substantial Losses Have Opportunity to Lead Investor Class Action - RGRD Law
Robbins Geller Rudman & Dowd LLP has announced an opportunity for investors of Ultragenyx Pharmaceutical Inc. who suffered substantial losses from August 2023 to December 2025 to lead a class action lawsuit. The lawsuit alleges violations of the Securities Exchange Act of 1934, citing misleading statements about the company’s product, setrusumab, particularly regarding the results of its Phase III Orbit study. On two separate occasions in 2025, Ultragenyx disclosed that this study, along with the Cosmic study, did not achieve statistical significance, resulting in significant stock price declines totaling more than 67%. Investors are encouraged to act by April 6, 2026, if they wish to serve as lead plaintiffs.
Read more →RAREUltragenyx Pharmaceutical Inc. Investors with Substantial Losses Have Opportunity to Lead Class Action Lawsuit - RGRD Law
Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit due to allegations of misleading statements regarding its Phase III Orbit study on Osteogenesis Imperfecta. The lawsuit claims that the company and its executives misrepresented their confidence in the study's results, which ultimately failed to demonstrate statistical significance. Following the disclosure of negative data from the study, Ultragenyx's stock price experienced significant declines, losing over 25% initially and 42% subsequently. Investors affected by these losses are now seeking to lead the class action against the company.
Read more →RAREUltragenyx to Participate at Investor Conferences in March 2026
Ultragenyx Pharmaceutical Inc. announced its participation in three investor conferences scheduled for March 2026. These include the Cowen Healthcare Conference in Boston, the Barclays Global Healthcare Conference, and the Leerink Partners Global Biopharma Conference, both in Miami. The company aims to highlight its commitment to developing therapies for serious rare and ultra-rare genetic diseases.
Read more →RAREUltragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Pharmaceutical Inc. announced the FDA's acceptance of their Biologics License Application for DTX401, a gene therapy targeting Glycogen Storage Disease Type Ia (GSDIa). The FDA has granted Priority Review, with a PDUFA action date set for August 23, 2026. If approved, DTX401 would be the first treatment to address the underlying cause of GSDIa, based on positive clinical trial results.
Read more →RAREUltragenyx Pharmaceutical Inc. Securities Class Action Filed; Lead Plaintiff Deadline April 6, 2026 – RGRD Law
A class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. for alleged securities violations during the period from August 3, 2023, to December 26, 2025. The lawsuit claims that the company misrepresented data regarding the Phase III Orbit study outcome, leading to significant stock price declines after the failure to achieve statistical significance in study results. Investors affected by this decline have until April 6, 2026, to seek appointment as lead plaintiff in the class action. The firm leading the lawsuit, Robbins Geller, is known for its significant recoveries in securities fraud cases.
Read more →RAREUltragenyx Pharmaceutical Inc. Sued for Securities Law Violations – Investors Should Contact Levi & Korsinsky for More Information – RARE
Levi & Korsinsky, LLP has initiated a class action lawsuit against Ultragenyx Pharmaceutical Inc. for alleged securities law violations. The lawsuit claims that the company misled investors about the efficacy of setrusumab, particularly regarding its relationship with fracture rates. Following the December 29, 2025 announcement that key studies did not meet their primary endpoints, Ultragenyx's stock price fell dramatically, declining approximately 42.32%. Investors potentially affected by the alleged fraud have until April 6, 2026, to seek class action participation.
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. has reported its fourth quarter and full year 2025 financial results, showcasing total revenues of $673 million, a 20% increase from the previous year. The company is implementing a strategic restructuring plan aimed at reducing operating expenses and aligning resources for a path to profitability by 2027. Significant future potential includes anticipated data readouts from pivotal Phase 3 studies and two possible product approvals in 2026. However, the company still faced a net loss of $575 million for the year and announced reductions in workforce.
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update 2025 to
Ultragenyx Pharmaceutical Inc. reported its financial results for the fourth quarter and full year ended December 31, 2025, showcasing a total revenue of $673 million, a 20% increase from the previous year. The company has initiated a strategic restructuring plan aimed at reducing expenses and headcount, expecting to achieve profitability by 2027. Looking ahead, Ultragenyx anticipates a revenue increase in 2026, with pivotal Phase 3 data for GTX-102 expected later in the year, alongside two potential product approvals.
Read more →RAREUltragenyx Pharmaceutical Inc. Securities Class Action Lawsuit Filed; Lead Plaintiff Deadline April 6, 2026 – RGRD Law
A securities class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc., alleging that the company made false statements regarding the efficacy of setrusumab in treating Osteogenesis Imperfecta. The lawsuit, which covers investors who acquired stock between August 3, 2023, and December 26, 2025, points to significant stock price declines following announcements of study result failures. Investors have until April 6, 2026, to seek lead plaintiff status in the case, which highlights concerns over the company's communication of clinical trial risks.
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2025 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will host a conference call on February 12, 2026, to discuss its financial results and corporate updates for Q4 and the full year of 2025. The call will be accessible via the company's website, with a replay available for three months. The company focuses on developing therapies for rare genetic diseases.
Read more →RAREULTRAGENYX ALERT: Bragar Eagel & Squire, P.C. Announces that a Class Action Lawsuit Has Been Filed Against Ultragenyx Pharmaceutical Inc. and Encourages Investors to Contact the Firm
Bragar Eagel & Squire, P.C. has announced a class action lawsuit against Ultragenyx Pharmaceutical Inc., related to losses suffered by investors who purchased shares within a specific period. The lawsuit targets the company's misleading statements regarding the effectiveness of setrusumab in its Phase III studies. It alleges that while Ultragenyx had projected positive outcomes, the studies fell short and did not achieve the desired statistical significance, leading to a substantial drop in stock price. Investors are encouraged to contact the firm for further legal recourse as the case develops.
Read more →RAREUltragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA)
Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA)
Read more →RAREUltragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA
Ultragenyx has resubmitted its Biologics License Application for UX111, a gene therapy for Sanfilippo syndrome type A, to the FDA. The submission includes long-term clinical data demonstrating positive effects and aims for accelerated approval. The FDA previously recognized the robustness of neurodevelopmental data, and if approved, UX111 will be the first treatment for this rare disease.
Read more →RAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) - January 23, 2026
Ultragenyx Pharmaceutical Inc. has granted 16,355 restricted stock units to nine new non-executive officers as part of its Employment Inducement Plan. This grant, approved by the board's compensation committee, is aimed at incentivizing new employees and aligns with Nasdaq Listing Rule 5635(c)(4). The stock units will vest over four years, contingent on continued employment.
Read more →RARE▲ +8.3%on this news
Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference
Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference
Read more →RARE▲ +7.4%on this newsshared move
Ultragenyx to Present at the 44th Annual J.P. Morgan Healthcare Conference
Ultragenyx Pharmaceutical Inc. will present at the 44th Annual J.P. Morgan Healthcare Conference on January 12, 2026. The company's CEO, Emil D. Kakkis, will lead the presentation which is aimed at enhancing the visibility of their novel therapies for rare genetic diseases. The presentation will be accessible via a live and archived webcast on Ultragenyx's website. Ultragenyx is known for its commitment to developing treatments for conditions with high unmet medical needs.
Read more →RAREUltragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investment
Ultragenyx has reported preliminary unaudited revenue for 2025, projecting totals between $672 million and $674 million, which exceeds initial guidance and showcases a growth of around 20% from the previous year. The company also holds approximately $735 million in cash and investments as of year-end 2025. Looking forward, Ultragenyx anticipates significant clinical and regulatory developments, including potential approvals for gene therapies targeting rare genetic diseases. The upcoming year is viewed as a transformative period for the company and the diseases they address.
Read more →RARE▲ +15.5%on this newsshared move
Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Read more →RARE▲ +15.5%on this newsshared move
Ultragenyx Pharmaceutical, Inc. Investigated by the Portnoy Law Firm
Ultragenyx Pharmaceutical, Inc. is under investigation by the Portnoy Law Firm for potential securities fraud after the company announced that both its Phase III Orbit and Cosmic studies failed to meet their primary endpoints in evaluating setrusumab, leading to a substantial drop in stock price. Following the disclosure, the share price fell by 43.49%, hitting a new 52-week low. Additionally, Ultragenyx revealed plans for significant expense reductions due to these setbacks. Investors are being encouraged to explore their legal options regarding the situation.
Read more →RARE▼ -42.3%on this newsshared move
Ultragenyx Announces Phase 3 Orbit and Cosmic Results for Setrusumab (UX143) in Osteogenesis Imperfecta
Ultragenyx announced that its Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta did not meet primary endpoints for reducing fracture rates. However, both studies achieved secondary endpoints showing improvements in bone mineral density. The company plans to analyze the data further and implement expense reductions in light of the results.
Read more →RAREUltragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) - December 19, 2025
Ultragenyx Pharmaceutical Inc. has granted 13,144 restricted stock units to nine newly hired non-executive officers as part of its Employment Inducement Plan. This grant, approved by the company's compensation committee, is intended to attract talent in line with Nasdaq Listing Rule 5635(c)(4). The stock units will vest over four years, contingent on continued employment.
Read more →RAREUltragenyx to Participate in Investor Conferences in December
Ultragenyx Pharmaceutical Inc. announced its participation in two investor conferences in December 2025. The conferences include Citi’s 2025 Global Healthcare Conference in Miami and the 8th Annual Evercore ISI Healthcare Conference in Coral Gables. The company aims to showcase its commitment to developing therapies for rare and ultra-rare genetic diseases.
Read more →RAREUltragenyx Reports Third Quarter 2025 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. reported a total revenue of $160 million for Q3 2025, marking a 15% increase from the previous year. The company reaffirmed its 2025 revenue guidance, expecting total revenues between $640 million and $670 million. Notably, Ultragenyx secured $400 million through the sale of a portion of its Crysvita royalty interest, enhancing its financial position ahead of significant clinical milestones. However, the company also reported a net loss of $180 million for the quarter.
Read more →RAREUltragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita® (burosumab) for $400 Million to OMERS Life Sciences
Ultragenyx Pharmaceutical has announced a $400 million sale of a portion of its future North American royalties on Crysvita to OMERS Life Sciences. This agreement will provide Ultragenyx with non-dilutive capital and is expected to bolster its balance sheet as it prepares for four anticipated product launches. OMERS will receive an additional 25% royalty interest starting in January 2028, capped at 1.55 times the purchase price.
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update Third quarter total revenue of $160 million, Crysvita revenue o
Ultragenyx Pharmaceutical Inc. reported its financial results for the third quarter of 2025, indicating a total revenue of $160 million, which reflects a 15% increase from the previous year. The company reaffirmed its 2025 revenue guidance, projecting total revenues between $640 million and $670 million along with a net loss of $180 million for the quarter. Significant strides were made in the clinical pipeline, with several anticipated data readouts and regulatory submissions expected in the near future. The company is also focusing on enhancing its financial position through strategic royalty sales.
Read more →RAREUltragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups
Ultragenyx Pharmaceutical has announced the dosing of the first patient in the Aurora study, which evaluates GTX-102 for Angelman syndrome. This study aims to include a wider age range and various genotypes not covered in the previous Phase 3 Aspire study. The Aurora trial reflects a commitment to expanding treatment opportunities for the Angelman syndrome community.
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2025 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will host a conference call on November 4, 2025, at 5:00 p.m. ET to discuss its financial results and corporate updates for Q3 2025. The call will be accessible via the company's website, with a replay available for three months. The company focuses on developing therapies for rare genetic diseases.
Read more →RAREUltragenyx annuncia l'approvazione da parte dell'AIFA (Agenzia Italiana del Farmaco) del rimborso di Evkeeza® (evinacumab) per i pazienti pediatrici di età pari a 6 mesi con ipercolesterolemia familiare omozigote (HoFH)
Ultragenyx Pharmaceutical Inc. announced that the Italian Medicines Agency (AIFA) has approved the reimbursement of Evkeeza (evinacumab) for pediatric patients aged 6 months and older with homozygous familial hypercholesterolemia (HoFH). This approval marks a significant advancement in treatment options for this rare genetic condition, which can lead to severe cardiovascular issues if untreated.
Read more →RAREUltragenyx Appoints Eric Olson as Chief Business Officer and Executive Vice President
Ultragenyx Pharmaceutical Inc. has appointed Eric Olson as Chief Business Officer and Executive Vice President, effective September 22, 2025. Olson succeeds Thomas Kassberg, who is retiring after 14 years. With extensive experience in business development, Olson aims to enhance Ultragenyx's mission to deliver new therapies for rare diseases.
Read more →RAREUltragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Pharmaceutical Inc. announced positive long-term results from its Phase 3 study of DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa). At Week 96, participants showed a 61% reduction in daily cornstarch intake while maintaining glycemic control. The therapy was well tolerated, with no serious adverse effects reported. These findings suggest DTX401 may significantly improve the management of GSDIa.
Read more →RAREUltragenyx to Participate at Investor Conferences in September
Ultragenyx to Participate at Investor Conferences in September
Read more →RAREUltragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
Read more →RAREUltragenyx Reports Second Quarter 2025 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Second Quarter 2025 Financial Results and Corporate Update Second quarter total revenue of $166 million, Crysvita revenue
Ultragenyx Pharmaceutical Inc. reported its financial results for the second quarter of 2025, achieving total revenue of $166 million, a 13% increase from the same period in 2024. Key contributors to growth included Crysvita and Dojolvi, with revenues of $120 million and $23 million respectively. The company reaffirmed its 2025 revenue guidance and continues to work towards profitability by 2027. Significant developments include GTX-102 receiving Breakthrough Therapy Designation and progress in ongoing clinical studies for UX143 and UX111, although the latter faces regulatory challenges from the FDA.
Read more →RAREUltragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Second Quarter 2025 Financial Results and Corporate Update
Read more →RAREUltragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RARE▲ +5.1%on this news· ran to -26% by day 3shared move
Ultragenyx and Mereo BioPharma Announce UX143 Phase 3 Orbit Study for Osteogenesis Imperfecta Progressing to Final Analysis
Ultragenyx Pharmaceutical and Mereo BioPharma have announced that their Phase 3 Orbit study for UX143 (setrusumab) in patients with osteogenesis imperfecta is progressing toward final analysis, expected later this year. The Data Monitoring Committee has confirmed an acceptable safety profile for UX143, encouraging the continuation of the study. Both the Orbit and Cosmic studies aim to evaluate the treatment's effectiveness in increasing bone mass and reducing fractures in pediatric and young adult patients, filling a significant gap in current treatment options for osteogenesis imperfecta.
Read more →RAREUltragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Read more →RAREUltragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
Ultragenyx to Participate at Goldman Sachs 46th Annual Global Healthcare Conference
Read more →RAREUltragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report
Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report
Read more →RAREUltragenyx to Participate at Bank of America’s 2025 Healthcare Conference
Ultragenyx Pharmaceutical Inc. has announced that its CFO, Howard Horn, will participate in a fireside chat at Bank of America's 2025 Healthcare Conference scheduled for May 13, 2025. This event aims to showcase the company's commitment to rare disease therapeutics. Ultragenyx focuses on developing novel therapies for serious rare and ultra-rare genetic diseases, emphasizing their strategy for efficient drug development. The event will be accessible via a live and archived webcast on the company's website.
Read more →RARE▼ -9.8%on this newsshared move
Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update First quarter total revenue of $139 million, Crysvita revenue o
Ultragenyx Pharmaceutical Inc. reported its financial results for Q1 2025, marking a total revenue of $139 million, a notable 28% growth year-over-year. The company reaffirmed its 2025 financial guidance, expecting revenues between $640 million and $670 million and highlighting growth in Crysvita and Dojolvi sales. Ultragenyx is advancing its investigational treatments, including UX111 for Sanfilippo syndrome, which is undergoing FDA review with an action date set for August 18, 2025. The ongoing clinical trials for its therapies also show promising results.
Read more →RARE▼ -9.8%on this newsshared move
Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2025 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. has announced a conference call scheduled for May 6, 2025, to discuss its financial results and corporate updates for Q1 2025. The call is set to take place at 5:00 p.m. ET, with live and replay options available via the company's website. Ultragenyx specializes in developing treatments for rare genetic diseases and aims to provide effective therapies for unmet medical needs.
Read more →RAREUltragenyx to Participate at Investor Conferences in March
Ultragenyx to Participate at Investor Conferences in March
Read more →RAREUltragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update 2024 Total Revenue of $560 million, exceeding gu
Ultragenyx Pharmaceutical Inc. reported its fourth-quarter and full-year financial results for 2024, showcasing total revenue of $560 million, which surpassed guidance. The company indicated expected total revenue growth of 14-20% for 2025, driven by products such as Crysvita and Dojolvi. However, Ultragenyx also noted a substantial net loss of $569 million for the year. Key upcoming events include potential regulatory decisions for its gene therapies in Sanfilippo syndrome and Glycogen Storage Disease Type Ia, alongside pivotal trial results for osteogenesis imperfecta and Angelman syndrome.
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. reported its financial results for Q4 and full year 2024, showcasing a total revenue of $560 million, a 29% growth compared to 2023. The company anticipates revenue growth to continue into 2025, projecting between $640 million and $670 million. Despite strong product sales, particularly from Crysvita and Dojolvi, Ultragenyx recorded a significant net loss of $133 million for the fourth quarter. Upcoming regulatory decisions and clinical trial results are expected to play a critical role in the company’s future performance.
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. announced that it will host a conference call on February 13, 2025, to discuss its financial results for the fourth quarter and full year ending December 31, 2024. The biopharmaceutical company, focused on treatments for rare genetic diseases, will provide updates on its corporate developments during the call. Interested parties can access the live and recorded webcasts through the company's website.
Read more →RAREUltragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS
Ultragenyx Pharmaceutical Inc. reported encouraging data for its UX111 AAV gene therapy in treating Sanfilippo syndrome Type A, showing substantial improvements in clinical function across cognitive domains in children. The treatment was correlated with significant reductions in CSF-HS levels, suggesting a robust therapeutic effect. Results from the pivotal Transpher A study indicated enhanced cognitive scores and communication abilities compared to untreated patients. This therapy aims to address a critical need in a devastating disorder, with safety data showing it was well-tolerated. The findings will be presented at the upcoming WORLDSymposium 2025.
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Carolyn Wang media@ultragenyx.com Ult ragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 202
Ultragenyx Pharmaceutical Inc. has reported preliminary 2024 revenues of $555 million to $560 million, surpassing prior guidance and reflecting robust growth. The company anticipates total revenues between $640 million and $670 million for 2025. Key pipeline updates include advancements in the Phase 3 studies for treatments of osteogenesis imperfecta and Angelman syndrome, with a potential gene therapy launch expected in the near future. Ultragenyx is positioned to introduce several new therapies as it builds up to eight or nine products over the next decade.
Read more →RAREUltragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones
Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones
Read more →RAREUltragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference
Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference
Read more →RAREUltragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Read more →RAREUltragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RAREUltragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST's 17th Annual Global Science Summit
Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST's 17th Annual Global Science Summit
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update Third quarter 2024 total revenue grew 42% versus prior year to
Ultragenyx Pharmaceutical Inc. reported significant growth in its third-quarter financial results for 2024, with total revenue reaching $139 million, a 42% increase compared to the prior year. The company reaffirmed its guidance for full-year revenues between $530 million and $550 million. Notably, it received Breakthrough Designation for its drug setrusumab (UX143) aimed at treating osteogenesis imperfecta. Positive results from its Phase 3 study of DTX401 for glycogen storage disease type Ia were also shared, showing substantial improvements in patient treatment outcomes.
Read more →RAREUltragenyx Reports Third Quarter 2024 Financial Results and Corporate Update
Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2024 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Third Quarter 2024 Financial Results and Corporate Update
Read more →RAREUltragenyx Receives Breakthrough Therapy Designation for Setrusumab (UX143) in Osteogenesis Imperfecta
Ultragenyx Receives Breakthrough Therapy Designation for Setrusumab (UX143) in Osteogenesis Imperfecta
Read more →RAREUltragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Read more →RAREUltragenyx Announces Upcoming Setrusumab (UX143) Presentations at the ASBMR 2024 Annual Meeting
Ultragenyx Announces Upcoming Setrusumab (UX143) Presentations at the ASBMR 2024 Annual Meeting
Read more →RAREUltragenyx to Participate in Investor Conferences in September
Ultragenyx to Participate in Investor Conferences in September
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Second Quarter 2024 Financial Results and Corporate Update Second quarter total revenue of $147 million, Crysvita revenue
Ultragenyx Pharmaceutical Inc. reported its financial results for the second quarter of 2024, revealing a total revenue of $147 million, up 36% year-over-year. The company announced a revision in its total revenue guidance for 2024, increasing it to between $530 million and $550 million. Alongside these results, promising clinical trial data were shared for therapies targeting Angelman syndrome and osteogenesis imperfecta. Despite these achievements, Ultragenyx continued to face substantial net losses and high operating costs.
Read more →RAREUltragenyx Reports Second Quarter 2024 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2024 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2024 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will host a conference call on August 1, 2024, to discuss its financial results and corporate update for Q2 2024. The company specializes in developing and commercializing products for rare genetic diseases. This call will offer insights into their ongoing efforts to address unmet medical needs with their portfolio of therapies.
Read more →RAREUltragenyx to Present GTX-102 Angelman Syndrome Program Update at the ASF Family Conference and Research Symposium
Ultragenyx to Present GTX-102 Angelman Syndrome Program Update at the ASF Family Conference and Research Symposium
Read more →RAREUltragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program
Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program
Read more →RARE▼ -9.4%on this newsshared move
Ultragenyx Announces Pricing of Public Offering of Common Stock and Pre-Funded Warrants
Ultragenyx Pharmaceutical Inc. has announced the pricing of an underwritten public offering of 7,435,898 shares at $39.00 per share, alongside pre-funded warrants for 1,538,501 shares at $38.999 each. The company expects to raise approximately $350 million before expenses, with the offering targeted to close around June 17, 2024. The offering is being managed by major financial institutions, and it is subject to customary closing conditions. Ultragenyx remains committed to developing therapies for rare genetic diseases, although the offering presents certain market and execution risks.
Read more →RARE▲ +7.8%on this newsshared move
Ultragenyx Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants
Ultragenyx Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants
Read more →RARE▲ +7.8%on this newsshared move
Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RAREUltragenyx and Mereo BioPharma Announce New Phase 2 Data from Phase 2/3 Orbit Study Demonstrating Sustained Reductions in Fracture Rates Following Treatment with Setrusumab (UX143) in Patients with Osteogenesis Imperfecta (OI)
Ultragenyx and Mereo BioPharma Announce New Phase 2 Data from Phase 2/3 Orbit Study Demonstrating Sustained Reductions in Fracture Rates Following Treatment with Setrusumab (UX143) in Patients with Osteogenesis Imperfecta (OI)
Read more →RAREUltragenyx to Participate at Goldman Sachs 45th Annual Global Healthcare Conference
Ultragenyx to Participate at Goldman Sachs 45th Annual Global Healthcare Conference
Read more →RAREUltragenyx Announces Positive Top-Line Results from Phase 3 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Announces Positive Top-Line Results from Phase 3 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Read more →RAREUltragenyx to Participate at Bank of America's 2024 Healthcare Conference
Ultragenyx to Participate at Bank of America's 2024 Healthcare Conference
Read more →RAREUltragenyx Reports First Quarter 2024 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update First quarter total revenue of $109 million, Crysvita revenue o
Ultragenyx Pharmaceutical Inc. reported its first quarter 2024 financial results with total revenues of $109 million, an 8% increase compared to the previous year. Notably, Crysvita revenue reached $83 million, reflecting a 9% growth. The company reaffirmed its 2024 revenue guidance and highlighted positive interim data from the GTX-102 study for Angelman syndrome, which showed significant improvements in multiple domains. Ultragenyx also announced successful enrollment completion for its Phase 3 programs in osteogenesis imperfecta, maintaining strong interest from physicians and the patient community.
Read more →RAREUltragenyx Announces Completion of Enrollment in Phase 3 Orbit and Cosmic Studies Evaluating Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta (OI)
Ultragenyx Announces Completion of Enrollment in Phase 3 Orbit and Cosmic Studies Evaluating Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta (OI)
Read more →RAREUltragenyx Issues 2023 Corporate Responsibility Report, Showcasing Commitment to Improving Equity and Access to Innovation in Rare Disease
Ultragenyx Issues 2023 Corporate Responsibility Report, Showcasing Commitment to Improving Equity and Access to Innovation in Rare Disease
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2024 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for First Quarter 2024 Financial Results and Corporate Update
Read more →RARE▼ -8.8%on this news
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
Read more →RARE▼ -7.3%on this newsshared move
Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting
Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting
Read more →RARE▼ -7.3%on this newsshared move
Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome
Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update 2023 Total Revenue of $434 million, Crysvita rev
Ultragenyx Pharmaceutical Inc. reported financial results for the fourth quarter and full year of 2023, posting a total revenue of $434 million, up 20% from the previous year. Crysvita and Dojolvi contributed significantly to this growth. The company anticipates total revenue for 2024 to reach between $500 million and $530 million. Several clinical milestones are on the horizon for 2024, including data on ongoing trials in Angelman syndrome and Osteogenesis Imperfecta.
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2023 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2023 Financial Results and Corporate Update
Read more →RAREUltragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)
Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)
Read more →RAREUltragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome
Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome
Read more →RAREUltragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Carolyn Wang media@ultragenyx.com Ultragenyx Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Upda
Ultragenyx Pharmaceutical Inc. has reported preliminary unaudited revenue for 2023, totaling between $430 million to $435 million, and provided guidance for 2024, anticipating revenue between $500 million and $530 million. The company also highlighted important upcoming clinical milestones in 2024 related to various therapies in development. Their year-end cash position is approximately $776 million, with expected operational cash usage projected to be less than $400 million in 2024. Key Phase 3 studies across several programs are set to provide interim data and results within the year.
Read more →RAREUltragenyx Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Updates and 2024 Milestones
Ultragenyx Reports Preliminary 2023 Revenue; Guidance for 2024 Revenue and Cash Usage; Pipeline Updates and 2024 Milestones
Read more →RAREUltragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza (evinacumab) for Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)
Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza (evinacumab) for Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)
Read more →RAREUltragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome
Ultragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome
Read more →RAREUltragenyx to Present at the 42nd Annual J.P. Morgan Healthcare Conference
Ultragenyx to Present at the 42nd Annual J.P. Morgan Healthcare Conference
Read more →RAREUltragenyx Receives European Commission Decision for Evkeeza (evinacumab) Expanded Indication in Children Aged 5 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)
Ultragenyx Pharmaceutical Inc. announced that the European Commission has expanded the approval of Evkeeza (evinacumab) for children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). This makes Evkeeza the first treatment option for this age group to significantly lower dangerously high LDL-C levels. Positive Phase 3 trial results indicate an average 48% reduction in LDL-C in treated children. While the safety profile is consistent with prior approvals, it includes new side effects that warrant attention.
Read more →RAREUltragenyx to Participate at Investor Conferences in November
Ultragenyx to Participate at Investor Conferences in November
Read more →RAREUltragenyx to Participate in the Jefferies London Healthcare Conference
Ultragenyx to Participate in the Jefferies London Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Third Quarter 2023 Financial Results and Corporate Update Third quarter 2023 total
Ultragenyx Pharmaceutical Inc. reported total revenues of $98.1 million for Q3 2023, reflecting 8% growth from the prior year. The company reaffirmed its annual revenue guidance, expecting between $425 million to $450 million in total revenue for 2023. Key clinical developments were highlighted, showcasing promising data from UX143 for osteogenesis imperfecta, GTX-102 for Angelman syndrome, and UX701 for Wilson disease at an Analyst Day event in October. Despite a reported net loss of $159.6 million in Q3, the company noted strong financial positioning due to increased product demand and effective management strategies.
Read more →RAREUltragenyx Reports Third Quarter 2023 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. reported its financial results for the third quarter of 2023, showing total revenues of $98.1 million, an 8% growth compared to the previous year. The company reaffirmed its revenue guidance for the year, projecting total revenue between $425 million and $450 million and highlighting progress in its key clinical programs. Despite a net loss of $159.6 million, Ultragenyx showcased promising interim data from its lead therapies addressing osteogenesis imperfecta, Angelman syndrome, and Wilson disease, indicating potential future growth.
Read more →RARE▲ +5.1%on this news
Ultragenyx to Host Conference Call for Third Quarter 2023 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. will hold a conference call on November 2, 2023, at 5:00 p.m. ET to discuss its third-quarter 2023 financial results and provide a corporate update. The company specializes in developing therapies for rare genetic diseases and aims to address significant unmet medical needs. A live and recorded webcast of the call will be accessible on the company’s website, and the replay will be available for one year.
Read more →RAREUltragenyx Announces Closing of Public Offering of Common Stock and Pre-Funded Warrants and Full Exercise of Underwriters’ Option to Purchase Additional Shares
Ultragenyx Pharmaceutical Inc. has successfully closed its underwritten public offering of 9,833,334 shares of common stock, including an additional 1,500,000 shares purchased by underwriters. The offering generated net proceeds of about $326.1 million, which will support the company's ongoing commitment to developing therapies for rare diseases. The offering included pre-funded warrants for certain investors, demonstrating interest and confidence in Ultragenyx’s future endeavors. The transaction was managed by major financial institutions, highlighting its significance in the market.
Read more →RAREUltragenyx Announces Proposed Public Offering of Common Stock
Ultragenyx Announces Proposed Public Offering of Common Stock
Read more →RARE▲ +10.3%on this newsshared move
Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease
Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease
Read more →RARE▲ +10.3%on this newsshared move
Ultragenyx and Mereo BioPharma Announce Interim Phase 2 Data from Phase 2/3 Orbit Study Demonstrating Setrusumab (UX143) Significantly Reduced Fracture Rates in Patients with Osteogenesis Imperfecta (OI) Phase 2 data pre
Ultragenyx Pharmaceutical and Mereo BioPharma have announced positive interim data from the Phase 2 portion of their Phase 2/3 Orbit study on setrusumab (UX143) for Osteogenesis Imperfecta. The data demonstrated a 67% reduction in annualized fracture rates and improvements in lumbar spine bone mineral density among treated patients. No serious adverse events were reported, and ongoing studies are underway to evaluate the drug's efficacy further. The Phase 3 portion of the study is currently enrolling approximately 195 patients across 12 countries.
Read more →RAREUltragenyx to Present Setrusumab (UX143) Update at ASBMR 2023 Including New Data from Phase 2/3 Orbit Study in Osteogenesis Imperfecta (OI)
Ultragenyx Pharmaceutical Inc. will present new data on setrusumab (UX143) at the ASBMR 2023 Annual Meeting, focusing on its Phase 2/3 Orbit study in osteogenesis imperfecta (OI). The presentations will detail the clinical fracture rates in young patients and the overall burden of fractures associated with OI. Setrusumab is designed to inhibit sclerostin, potentially increasing bone strength and mineral density. The collaboration with Mereo BioPharma continues to expand the late-stage development of this novel therapy.
Read more →RAREUltragenyx to Present Pipeline Update at Analyst Day on Monday, October 16, 2023
Ultragenyx Pharmaceutical Inc. will hold an Analyst Day on October 16, 2023, to present updates on its development pipeline. The agenda will feature discussions on UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome. Notable experts, including principal investigators from prominent universities, will participate in panel discussions. A live webcast will be available for those interested in following the updates on their therapeutic advancements.
Read more →RAREUltragenyx Launches Evkeeza (evinacumab for injection) in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)
Ultragenyx Pharmaceutical Inc. has announced the approval of Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH) in Canada, effective November 25, 2023. This first-of-its-kind monoclonal antibody treatment is designed for both adult and pediatric patients aged 5 years and older and aims to significantly lower LDL cholesterol levels. The company emphasizes its dedication to innovative therapies for rare diseases while collaborating with healthcare providers for patient access. However, the effects of Evkeeza on cardiovascular health remain unconfirmed, and some risks exist regarding hypersensitivity reactions.
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Second Quarter 2023 Financial Results and Corporate Update Second quarter 2023 tota
Ultragenyx Pharmaceutical Inc. reported its financial results for Q2 2023, revealing total revenue of $108.3 million, a 21% increase from the same quarter in 2022. Crysvita and Dojolvi contributed significantly to this growth, with revenues of $83.0 million and $16.5 million, respectively. The company reaffirmed its expectation for total revenue in 2023 to be between $425 million and $450 million. Despite the revenue growth, Ultragenyx experienced a net loss of $159.8 million in the quarter.
Read more →RAREUltragenyx Reports Second Quarter 2023 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. reported a total revenue of $108.3 million for the second quarter of 2023, marking a 21% increase from the previous year. The company reaffirmed its full-year revenue guidance of $425 million to $450 million. Crysvita and Dojolvi segments demonstrated strong performance with revenues growing 20% and 22%, respectively. Key clinical programs for osteogenesis imperfecta and other conditions continue to advance, with significant data expected in the upcoming quarters. However, the company faced a net loss of $159.8 million during the quarter, raising questions about cost management and operational sustainability.
Read more →RAREUltragenyx Announces Initiation of Dosing in Second Cohort of Pivotal Phase 1/2/3 Cyprus2+ Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Ultragenyx Announces Initiation of Dosing in Second Cohort of Pivotal Phase 1/2/3 Cyprus2+ Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2023 Financial Results and Corporate Update
Ultragenyx Pharmaceutical Inc. has announced it will host a conference call on August 3, 2023, at 5:00 p.m. ET to discuss its financial results and provide a corporate update for the quarter ending June 30, 2023. This event demonstrates the company's commitment to transparency and communication with stakeholders. Ultragenyx continues to focus on developing treatments for serious rare and ultrarare genetic diseases, highlighting the company's urgency in delivering therapies with high unmet medical needs.
Read more →RAREUltragenyx Appoints Howard Horn as Chief Financial Officer and Executive Vice President, Corporate Strategy NOVATO, Calif. —
Ultragenyx Pharmaceutical Inc. has announced the appointment of Howard Horn as Chief Financial Officer and Executive Vice President, Corporate Strategy, effective October 16, 2023. Horn, who previously served as CFO at Vir Biotechnology, will oversee finance, accounting, corporate strategy, and investor relations as Ultragenyx prepares for significant growth. The company's pipeline is currently maturing, indicating a promising future in the rare disease space. Horn expressed enthusiasm for Ultragenyx's mission to aid patients with rare diseases.
Read more →RAREUltragenyx Announces First Patients Dosed in Phase 3 Program Evaluating Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta (OI) Pivotal Phase 3 portion of Orbit study now enrolling approximately 195 pediatri
Ultragenyx has initiated dosing in its Phase 3 clinical trials for Setrusumab, targeting pediatric and young adult patients with Osteogenesis Imperfecta (OI). The pivotal Phase 3 Orbit study will evaluate the drug's effect on clinical fracture rates, while the new Cosmic study will assess its effectiveness against intravenous bisphosphonate therapy in younger patients. The company aims to demonstrate clinical improvements based on encouraging results from prior studies, which indicated enhanced bone density and formation.
Read more →RAREUltragenyx Opens New Gene Therapy Manufacturing Facility in Bedford, Continuing its Expansion in Massachusetts
Ultragenyx Opens New Gene Therapy Manufacturing Facility in Bedford, Continuing its Expansion in Massachusetts
Read more →RAREUltragenyx to Participate in the Goldman Sachs 44th Annual Global Healthcare Conference
Ultragenyx Pharmaceutical Inc. announced its participation in the Goldman Sachs 44th Annual Global Healthcare Conference. CEO Emil D. Kakkis will present at the event on June 12, 2023. The webcast will be available on the company's website. Ultragenyx is committed to developing therapies for rare genetic diseases and has a portfolio focused on unmet medical needs.
Read more →RAREUltragenyx and Mereo BioPharma Announce Positive Data from the Ongoing Phase 2/3 Orbit Study of Setrusumab (UX143) in Osteogenesis Imperfecta (OI) Pediatric data show substantial induction of bone production in 1 week an
Ultragenyx and Mereo BioPharma reported encouraging results from the ongoing Phase 2/3 Orbit study evaluating their treatment setrusumab (UX143) in children with Osteogenesis Imperfecta. The data demonstrated significant increases in bone production and mineral density within three months of treatment initiation. Importantly, no serious adverse events were recorded during the study, suggesting a favorable safety profile for setrusumab. The Phase 3 portion is gearing up with plans to enroll approximately 195 patients across various countries.
Read more →RAREUltragenyx Issues 2022 Environmental, Social and Governance (ESG) Report
Ultragenyx Issues 2022 Environmental, Social and Governance (ESG) Report
Read more →RAREUltragenyx to Present at the 2023 Barclays - Gene Editing & Gene Therapy Summit
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company specializing in therapies for serious genetic diseases, announced that its chief medical officer, Eric Crombez, M.D., will present at the 2023 Barclays - Gene Editing & Gene Therapy Summit. The event is scheduled for May 24, 2023, and the presentation will be accessible via a live and archived webcast on the company's website. Ultragenyx aims to provide novel treatments for diseases with high unmet medical needs and has a diversified portfolio targeting these conditions.
Read more →RARE▲ +5.3%on this newsshared move
Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S.
Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S.
Read more →RAREUltragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting
Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting
Read more →RAREUltragenyx to Present at Bank of America's 2023 Health Care Conference
Ultragenyx to Present at Bank of America's 2023 Health Care Conference
Read more →RARE▲ +5.7%on this newsshared move
Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update First quarter 2023 t otal
Ultragenyx Pharmaceutical Inc. has reported a total revenue of $100.5 million for Q1 2023, marking a 26% increase from the prior year. Significant contributions came from the sales of Crysvita, which grew by 28%. The company reaffirmed its financial guidance for the year, expecting revenues between $425 million and $450 million. While the company experienced a net loss of $164 million, it is making progress in its clinical programs, including the advancement of UX143 in osteogenesis imperfecta and GTX-102 in Angelman syndrome.
Read more →RARE▲ +5.7%on this newsshared move
Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2023 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for First Quarter 2023 Financial Results and Corporate Update
Read more →RAREUltragenyx to Present at Guggenheim's Genomic Medicines and Rare Disease Days
Ultragenyx to Present at Guggenheim's Genomic Medicines and Rare Disease Days
Read more →RAREUltragenyx Appoints Dr. Eric Crombez Chief Medical Officer and Executive Vice President
Ultragenyx Appoints Dr. Eric Crombez Chief Medical Officer and Executive Vice President
Read more →RAREUltragenyx to Participate in Orphan Neuro Panel at Cowen Healthcare Conference
Ultragenyx to Participate in Orphan Neuro Panel at Cowen Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2022 Financial Results and Corporate Update 2022 Total
Ultragenyx Pharmaceutical Inc. reported its financial results for Q4 and the full year of 2022, showing total collaboration and product revenue of $355.6 million. For 2023, the company projects revenue between $425 million to $450 million, reflecting growth expectations. However, the company also faced a net loss of $707.4 million for the year, indicating ongoing financial challenges. The announcement also highlighted advancements in clinical programs targeting serious genetic diseases, with key data readouts expected in 2023.
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2022 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2022 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2022 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2022 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Preliminary 2022 Revenue; Guidance for 2023 Revenue and Cash Usage; Pipeline Update
Ultragenyx Pharmaceutical Inc. reported preliminary unaudited 2022 revenue results, with total product revenue between $352 million and $356 million, and provided guidance for 2023, expecting total revenue between $425 million to $450 million. The 2022 cash balance was approximately $900 million, with expected cash usage projected to be less than $400 million for 2023. The company highlighted several clinical milestones, including ongoing studies in treatments for Osteogenesis Imperfecta and Angelman Syndrome, positioning itself for significant advancements in addressing rare genetic diseases.
Read more →RAREUltragenyx Reports Preliminary 2022 Revenue; Guidance for 2023 Revenue and Cash Usage; Pipeline Updates and 2023 Milestones
Ultragenyx Reports Preliminary 2022 Revenue; Guidance for 2023 Revenue and Cash Usage; Pipeline Updates and 2023 Milestones
Read more →RAREUltragenyx to Present at the 41st Annual J.P. Morgan Healthcare Conference
Ultragenyx to Present at the 41st Annual J.P. Morgan Healthcare Conference
Read more →RAREUltragenyx to Present at Piper Sandler Healthcare Conference
Ultragenyx to Present at Piper Sandler Healthcare Conference
Read more →RARE▲ +11.4%on this news
Ultragenyx Announces Departure of Chief Financial Officer
Ultragenyx Announces Departure of Chief Financial Officer
Read more →RAREUltragenyx to Present at Upcoming Healthcare Conferences
Ultragenyx to Present at Upcoming Healthcare Conferences
Read more →RAREUltragenyx Reports Third Quarter 2022 Financial Results and Corporate Update
Ultragenyx Reports Third Quarter 2022 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Third Quarter 2022 Financial Results and Corporate Update Third quarter 2022 total
Contacts Ultragenyx Pharmaceutical Inc. media@ultragenyx.com Ultragenyx Reports Third Quarter 2022 Financial Results and Corporate Update Third quarter 2022 total revenue of $90.7 million and Crysvita revenue in Ultragenyx territories1 of $64.5 million Reaffirm 2022 Crysvita
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2022 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Third Quarter 2022 Financial Results and Corporate Update
Read more →RAREUltragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR
Ultragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR
Read more →RAREUltragenyx to Participate at Citi BioPharma Conference
Ultragenyx to Participate at Citi BioPharma Conference
Read more →RAREUltragenyx Reports Second Quarter 2022 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2022 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Second Quarter 2022 Financial Results and Corporate Update Second quarter 2022 tota
Contacts Ultragenyx Pharmaceutical Inc. media@ultragenyx.com Ultragenyx Reports Second Quarter 2022 Financial Results and Corporate Update Second quarter 2022 total revenue of $89.3 million and Crysvita revenue in Ultragenyx territories1 of $64.0 million Reaffirm 2022 Crysvit
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2022 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Second Quarter 2022 Financial Results and Corporate Update
Read more →RARE▼ -5.8%on this news· ran to -18% by day 1shared move
Ultragenyx and GeneTx Provide Program Update on GTX-102 for Angelman Syndrome Including Promising Interim Data from Phase 1/2 Study Doses up to 10 mg show good tolerability and meaningful clinical activity in multiple do
Ultragenyx and GeneTx Provide Program Update on GTX-102 for Angelman Syndrome Including Promising Interim Data from Phase 1/2 Study Doses up to 10 mg show good tolerability and meaningful clinical activity in multiple domains U.K. and Canadian health authorities approved escala
Read more →RAREUltragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $500 Million to OMERS Capital Markets Strengthens balance sheet with non-dilutive capital at an attractive cost Compet
Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $500 Million to OMERS Capital Markets Strengthens balance sheet with non-dilutive capital at an attractive cost Competitive process results in the sale of 30% of the Ultragenyx
Read more →RAREUltragenyx to Present at Goldman Sachs Global Healthcare Conference
Ultragenyx to Present at Goldman Sachs Global Healthcare Conference
Read more →RAREUltragenyx to Present at Jefferies Global Healthcare Conference
Ultragenyx to Present at Jefferies Global Healthcare Conference
Read more →RAREUltragenyx Announces Positive Longer-term Durability Data from Two Phase 1/2 Gene Therapy Studies at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
Ultragenyx Announces Positive Longer-term Durability Data from Two Phase 1/2 Gene Therapy Studies at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
Read more →RAREUltragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics
Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics
Read more →RARE▼ -10%on this news
Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
NOVATO, Calif., May 09, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that clinical, preclinical and manufact
Read more →RARE▼ -6.3%on this news· ran to -24% by day 3shared move
Ultragenyx Reports First Quarter 2022 Financial Results and Corporate Update
First quarter 2022 total revenue of $79.9 million and Crysvita® revenue in Ultragenyx territories1 of $54.6 million Reaffirm 2022 Crysvita revenue in Ultragenyx territories guidance of $250 million to $260 million and Dojolvi revenue of $55 million to $65 million Cash balance o
Read more →RARE▼ -6.3%on this news· ran to -24% by day 3shared move
Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics ABO-102 / UX111 is being evaluated in ongoing pivotal Transpher A trial in patients with MP
Ultragenyx Acquires Global Rights to AAV Gene Therapy ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) from Abeona Therapeutics ABO-102 / UX111 is being evaluated in ongoing pivotal Transpher A trial in patients with MPS IIIA Interim data featured in encore oral presentation a
Read more →RARE▼ -6.3%on this news· ran to -24% by day 3shared move
Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports First Quarter 2022 Financial Results and Corporate Update First quarter 2022 total
Contacts Ultragenyx Pharmaceutical Inc. media@ultragenyx.com Ultragenyx Reports First Quarter 2022 Financial Results and Corporate Update First quarter 2022 total revenue of $79.9 million and Crysvita revenue in Ultragenyx territories1 of $54.6 million Reaffirm 2022 Crysvita
Read more →RAREUltragenyx to Participate at BofA Securities 2022 Healthcare Conference
NOVATO, Calif., May 03, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Camille Bedrosian
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2022 Financial Results and Corporate Update
NOVATO, Calif., April 27, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a c
Read more →RAREUltragenyx Issues Inaugural Environmental, Social and Governance (ESG) Report
NOVATO, Calif., April 25, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today issued its inaugural ESG report
Read more →RAREUltragenyx Appoints Amrit Ray, M.D., M.B.A. to Board of Directors
Ultragenyx Appoints Amrit Ray, M.D., M.B.A. to Board of Directors
Read more →RAREUltragenyx Announces First Patient Dosed in Pivotal Phase 2/3 Clinical Study of Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta
Ultragenyx Announces First Patient Dosed in Pivotal Phase 2/3 Clinical Study of Setrusumab (UX143) for the Treatment of Osteogenesis Imperfecta
Read more →RAREUltragenyx to Participate in the Cowen Health Care Conference
NOVATO, Calif., March 02, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis,
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jeff Blake media@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update 2021 total
Contacts Ultragenyx Pharmaceutical Inc. media@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update 2021 total revenue of $351.4 million and 2021 Crysvita revenue in Ultragenyx territories1 of $192.6 million 2022 Crysvita r
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2021 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2021 Financial Results and Corporate Update
Read more →RAREULTRAGENYX REPORTS PRELIMINARY 2021 REVENUE AND 2022 REVENUE GUIDANCE FOR CRYSVITA IN ULTRAGENYX TERRITORIES* AND DOJOLVI GLOBALLY; PROVIDES PIPELINE UPDATES AND 2022 MILESTONES Preliminary 2021 Crysvita revenue in Ultra
ULTRAGENYX REPORTS PRELIMINARY 2021 REVENUE AND 2022 REVENUE GUIDANCE FOR CRYSVITA IN ULTRAGENYX TERRITORIES* AND DOJOLVI GLOBALLY; PROVIDES PIPELINE UPDATES AND 2022 MILESTONES Preliminary 2021 Crysvita revenue in Ultragenyx territories of $191 million to $193 million and Dojol
Read more →RARE▼ -7.1%on this newsshared move
Regeneron and Ultragenyx Collaborate to Commercialize Evkeeza (evinacumab) Outside the United States Evkeeza is a first-in-class medicine approved by the U.S. Food and Drug Administration (FDA) and European
Regeneron and Ultragenyx Collaborate to Commercialize Evkeeza (evinacumab) Outside the United States Evkeeza is a first-in-class medicine approved by the U.S. Food and Drug Administration (FDA) and European Commission (EC) to treat an ultra-rare inherited form of high cholestero
Read more →RARE▼ -7.1%on this newsshared move
Regeneron and Ultragenyx Collaborate to Commercialize Evkeeza (evinacumab) Outside the United States
Regeneron and Ultragenyx Collaborate to Commercialize Evkeeza (evinacumab) Outside the United States
Read more →RAREUltragenyx to Present at H.C. Wainwright BioConnect Healthcare Conference
NOVATO, Calif., Jan. 06, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis,
Read more →RAREGeneTx and Ultragenyx Provide Preliminary Update on Phase 1/2 Clinical Study of GTX-102 in Canada and U.K. Patients with Angelman Syndrome
GeneTx and Ultragenyx Provide Preliminary Update on Phase 1/2 Clinical Study of GTX-102 in Canada and U.K. Patients with Angelman Syndrome
Read more →RAREUltragenyx to Present at 40th Annual JP Morgan Healthcare Conference
Ultragenyx to Present at 40th Annual JP Morgan Healthcare Conference
Read more →RAREUltragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III
Ultragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III
Read more →RAREGeneTx and Ultragenyx Announce Presentations at Upcoming 2021 FAST Global Summit & Gala
GeneTx and Ultragenyx Announce Presentations at Upcoming 2021 FAST Global Summit & Gala
Read more →RAREUltragenyx Announces Additional Positive Multi-Year Durability Data from Phase 1/2 AAV Gene Therapy Studies
Ultragenyx Announces Additional Positive Multi-Year Durability Data from Phase 1/2 AAV Gene Therapy Studies
Read more →RAREUltragenyx to Present at Evercore 4th Annual HealthconX Conference
NOVATO, Calif., Nov. 29, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis,
Read more →RAREUltragenyx Reports Third Quarter 2021 Financial Results and Corporate Update
Third quarter 2021 total revenue of $81.6 million including Crysvita1 revenue to Ultragenyx of $50.3 million 2021 Crysvita revenue in Ultragenyx territories1 now expected towards the upper end of the$180 million to $190 million range previously provided NOVATO, Calif., Nov. 02,
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa (415) 475-6370 ir@ultragenyx.com Media Carolyn Wang 415-225-5050 media@ultragenyx.com Ultragenyx Reports Third Quarter 2021 Financial Results and Corporate Upd
Contact Ultragenyx Pharmaceutical Inc. media@ultragenyx.com Ultragenyx Reports Third Quarter 2021 Financial Results and Corporate Update Third quarter 2021 total revenue of $81.6 million including Crysvita1 revenue to Ultragenyx of $50.3 million 2021 Crysvita revenue in Ultra
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2021 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Third Quarter 2021 Financial Results and Corporate Update
Read more →RAREUltragenyx to Present at Jefferies Gene Therapy/Editing Summit
Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit
Read more →RAREUltragenyx Initiates Cyprus2+, a Pivotal Clinical Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
NOVATO, Calif., Oct. 18, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare genetic diseases, today announced that it has successfully scree
Read more →RAREGeneTx and Ultragenyx Announce First Patient Dosed in Canada in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome SARASOTA, Fla., and NOVATO, Calif. —
GeneTx and Ultragenyx Announce First Patient Dosed in Canada in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome SARASOTA, Fla., and NOVATO, Calif. October 7, 2021 GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partn
Read more →RAREMereo BioPharma and Ultragenyx Present Data from the Phase 2b ASTEROID Study of UX143 (setrusumab) in Osteogenesis Imperfecta (OI) at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting
Mereo BioPharma and Ultragenyx Present Data from the Phase 2b ASTEROID Study of UX143 (setrusumab) in Osteogenesis Imperfecta (OI) at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting
Read more →RARE▼ -6.8%on this news
Ultragenyx Announces Crysvita (burosumab) and UX143 (setrusumab) Data Presentations at Upcoming American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting
Ultragenyx Announces Crysvita (burosumab) and UX143 (setrusumab) Data Presentations at Upcoming American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting
Read more →RARE▼ -6.8%on this news
GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1/2 Clinical Study of GTX-102 for the Treatment of Angelman Syndrome in the U.S. SARASOTA, Fla. and NOVATO, Calif —
GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1/2 Clinical Study of GTX-102 for the Treatment of Angelman Syndrome in the U.S. SARASOTA, Fla. and NOVATO, Calif September 27, 2021 GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)
Read more →RAREUltragenyx to Present at Morgan Stanley Healthcare Conference
NOVATO, Calif., Sept. 07, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis,
Read more →RAREUltragenyx to Participate in Panel at Citi's 16th Annual BioPharma Healthcare Conference
Ultragenyx to Participate in Panel at Citi's 16th Annual BioPharma Healthcare Conference
Read more →RARE▲ +5.6%on this news
Ultragenyx Announces Approval of Dojolvi (triheptanoin) in Brazil for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
Ultragenyx Announces Approval of Dojolvi (triheptanoin) in Brazil for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
Read more →RAREUltragenyx to Participate in Gene Therapy Panel at Wedbush PacGrow Healthcare Conference
Ultragenyx to Participate in Gene Therapy Panel at Wedbush PacGrow Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors Joshua Higa (415) 475-6370 Media Carolyn Wang 415-225-5050 Ultragenyx Reports Second Quarter 2021 Financial Results and Corporate Update Second quarter 2021 total revenue
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2021 Financial Results and Corporate Update Second quarter 2021 total revenue of $87.0 million including Crysvita1 revenue to Ultragenyx of $44.7 million 2021 Crysvita revenue in Ultragenyx territories1 g
Read more →RAREUltragenyx Reports Second Quarter 2021 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2021 Financial Results and Corporate Update
Read more →RAREUltragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III
Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2021 Financial Results and Corporate Update
NOVATO, Calif., July 27, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel therapies for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Mond
Read more →RAREUltragenyx Appoints Corsee Sanders, Ph.D., to Board of Directors
Ultragenyx Appoints Corsee Sanders, Ph.D., to Board of Directors
Read more →RAREGeneTx and Ultragenyx Receive Approval from U.K. Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome Patient enrollment to begin in early second half of 2021 in U.K. and Canada Gen
GeneTx and Ultragenyx Receive Approval from U.K. Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome Patient enrollment to begin in early second half of 2021 in U.K. and Canada GeneTx to submit revised protocol to U.S. Food and Drug Admin
Read more →RAREUltragenyx to Present at Goldman Sachs 42nd Annual Healthcare Conference
Ultragenyx to Present at Goldman Sachs 42nd Annual Healthcare Conference
Read more →RAREUltragenyx to Present at Jefferies Virtual Healthcare Conference
Ultragenyx to Present at Jefferies Virtual Healthcare Conference
Read more →RAREGeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome SARASOTA, Fla. and NOVATO, Calif —
GeneTx and Ultragenyx Receive Clearance from Health Canada to Begin Clinical Study of GTX-102 in Canada for the Treatment of Angelman Syndrome SARASOTA, Fla. and NOVATO, Calif May 19, 2021 GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies pa
Read more →RAREUltragenyx Announces Positive Multi-Year Durability Data from Ongoing Phase 1/2 Gene Therapy Studies and Data on HeLa 3.0 Manufacturing Platform at American Society of Gene & Cell Therapy 2021 Annual Meeting Durable and
Ultragenyx Announces Positive Multi-Year Durability Data from Ongoing Phase 1/2 Gene Therapy Studies and Data on HeLa 3.0 Manufacturing Platform at American Society of Gene & Cell Therapy 2021 Annual Meeting Durable and Clinically Meaningful Responses Maintained at More than 2.
Read more →RAREUltragenyx to Present at Bank of America Securities Healthcare Conference
Ultragenyx to Present at Bank of America Securities Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Joshua Higa (415) 475-6370 Ultragenyx Reports First Quarter 2021 Financial Results and Corporate Update First quarter 2021 total revenue of $99.4 million and Crysv
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2021 Financial Results and Corporate Update First quarter 2021 total revenue of $99.4 million and Crysvita1 revenue to Ultragenyx of $42.1 million 2021 Crysvita revenue in Ultragenyx territories1 guidance
Read more →RAREUltragenyx Reports First Quarter 2021 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2021 Financial Results and Corporate Update
Read more →RAREUltragenyx to Present at Truist Securities Life Sciences Summit
Ultragenyx to Present at Truist Securities Life Sciences Summit
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2021 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for First Quarter 2021 Financial Results and Corporate Update
Read more →RAREUltragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy 2021 Virtual Annual Meeting
Ultragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy 2021 Virtual Annual Meeting
Read more →RAREUltragenyx Completes Successful End-of-Phase 2 Meeting with FDA and Finalizes Phase 3 Study Design for DTX301 Ornithine Transcarbamylase (OTC) Gene Therapy Program
Ultragenyx Completes Successful End-of-Phase 2 Meeting with FDA and Finalizes Phase 3 Study Design for DTX301 Ornithine Transcarbamylase (OTC) Gene Therapy Program
Read more →RAREUltragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of Glycogen Storage Disease Type III
Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of Glycogen Storage Disease Type III
Read more →RAREUltragenyx to Present at Barclays Global Healthcare Conference
Ultragenyx to Present at Barclays Global Healthcare Conference
Read more →RAREUltragenyx Announces Approval of Dojolvi (UX007/triheptanoin) in Canada for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
Ultragenyx Announces Approval of Dojolvi (UX007/triheptanoin) in Canada for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children
Read more →RAREUltragenyx to Present at Upcoming Investor Conferences
Ultragenyx to Present at Upcoming Investor Conferences
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Joshua Higa (415) 475-6370 Ultragenyx Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Update 2020 total revenue of $271.0 million and 202
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Update 2020 total revenue of $271.0 million and 2020 Crysvita1 revenue to Ultragenyx of $138.9 million 2021 Crysvita1 revenue in Ultragenyx territories g
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2020 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2020 Financial Results and Corporate Update
Read more →RAREUltragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701, a New Gene Therapy for the Treatment of Wilson Disease Clinical trial will utilize a single-protocol Phase 1/2/3 design UX701 ma
Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701, a New Gene Therapy for the Treatment of Wilson Disease Clinical trial will utilize a single-protocol Phase 1/2/3 design UX701 manufacturing complete at commercial quality and scale using
Read more →RAREUltragenyx Reports Preliminary 2020 Revenue and 2021 Revenue Guidance for Crysvita in Ultragenyx Territories Preliminary 2020 Crysvita revenue in Ultragenyx Territories of $137 million to $139 million 2021 Crysvita Ultra
Ultragenyx Reports Preliminary 2020 Revenue and 2021 Revenue Guidance for Crysvita in Ultragenyx Territories Preliminary 2020 Crysvita revenue in Ultragenyx Territories of $137 million to $139 million 2021 Crysvita Ultragenyx revenue expected in the range of $180 million to $1
Read more →RAREUltragenyx Investors & Media Joshua Higa (415) 475-6370 Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies Durable and Cli
Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies Durable and Clinically Meaningful Responses Reported from Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC Phase 3 Studies for DT
Read more →RAREUltragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies
Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies
Read more →RAREUltragenyx to Present at 39th Annual J.P. Morgan Virtual Healthcare Conference
Ultragenyx to Present at 39th Annual J.P. Morgan Virtual Healthcare Conference
Read more →RAREUltragenyx and Mereo BioPharma Announce Collaboration and License Agreement for Setrusumab in Osteogenesis Imperfecta Clinical-stage monoclonal antibody in development for rare genetic bone disease that builds on Ultrage
Ultragenyx and Mereo BioPharma Announce Collaboration and License Agreement for Setrusumab in Osteogenesis Imperfecta Clinical-stage monoclonal antibody in development for rare genetic bone disease that builds on Ultragenyx's existing bone franchise Ultragenyx leads and funds d
Read more →RAREUltragenyx Announces Orphan Drug Designation for UX701 for the Treatment of Wilson Disease
Ultragenyx Announces Orphan Drug Designation for UX701 for the Treatment of Wilson Disease
Read more →RAREGeneTx and Ultragenyx Announce Presentation of Phase 1/2 Data on Investigational GTX-102 in Patients with Angelman Syndrome Results presented at Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit Addition
GeneTx and Ultragenyx Announce Presentation of Phase 1/2 Data on Investigational GTX-102 in Patients with Angelman Syndrome Results presented at Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit Additional data, including EEG findings, support prior initial ind
Read more →RAREUltragenyx Announces Plans to Build Large-scale Gene Therapy Manufacturing Facility to Support Pipeline of Therapies for Rare Diseases
Ultragenyx Announces Plans to Build Large-scale Gene Therapy Manufacturing Facility to Support Pipeline of Therapies for Rare Diseases
Read more →RAREUltragenyx Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters' Option to Purchase Additional Shares
Ultragenyx Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters' Option to Purchase Additional Shares
Read more →RAREUltragenyx Announces Pricing of Public Offering of Common Stock
Ultragenyx Announces Pricing of Public Offering of Common Stock
Read more →RAREUltragenyx Reports Third Quarter 2020 Financial Results and Corporate Update
Ultragenyx Reports Third Quarter 2020 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Joshua Higa (415) 475-6370 Ultragenyx Reports Third Quarter 2020 Financial Results and Corporate Update Third quarter 2020 total revenue of $81.5 million and Crysv
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Third Quarter 2020 Financial Results and Corporate Update Third quarter 2020 total revenue of $81.5 million and Crysvita1 revenue of $37.3 million Increased lower end of Crysvita1 guidance range; updated full year range
Read more →RAREGeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome Efficacy in multiple functional measures observed in all five patient
GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome Efficacy in multiple functional measures observed in all five patients treated Highest doses associated with a significant but
Read more →RAREUltragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy -Collaboration combines Solid's differentiated microdystrophin construct a
Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy -Collaboration combines Solid's differentiated microdystrophin construct and Ultragenyx's HeLa PCL manufacturing platform for use wit
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2020 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Third Quarter 2020 Financial Results and Corporate Update
Read more →RAREUltragenyx Appoints Mardi C. Dier as Chief Financial Officer
Ultragenyx Appoints Mardi C. Dier as Chief Financial Officer
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Joshua Higa (415) 475-6370 Ultragenyx Appoints Mardi C. Dier as Chief Financial Officer Ms. Dier will start in new role in November 2020 Novato, Calif. —
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Appoints Mardi C. Dier as Chief Financial Officer Ms. Dier will start in new role in November 2020 Novato, Calif. September 2, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the devel
Read more →RAREUltragenyx Expands Leadership Team and Appoints Ernie Meyer as Chief Human Resources Officer
Ultragenyx Expands Leadership Team and Appoints Ernie Meyer as Chief Human Resources Officer
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Joshua Higa (415) 475-6370 Ultragenyx Reports Second Quarter 2020 Financial Results and Corporate Update Two U.S. FDA approvals in second quarter 2020 Second quart
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2020 Financial Results and Corporate Update Two U.S. FDA approvals in second quarter 2020 Second quarter 2020 total revenue of $61.7 million and Crysvita revenue in Ultragenyx territories of $32.4 millio
Read more →RAREUltragenyx Reports Second Quarter 2020 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2020 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2020 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Second Quarter 2020 Financial Results and Corporate Update
Read more →RAREUltragenyx Announces U.S. Commercial Launch of Dojolvi (triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders
Ultragenyx Announces U.S. Commercial Launch of Dojolvi (triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders
Read more →RAREUltragenyx Announces U.S. FDA Approval of Dojolvi (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders Fourth U.S. FDA Approval for Ultragenyx in the Past Th
Ultragenyx Announces U.S. FDA Approval of Dojolvi (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders Fourth U.S. FDA Approval for Ultragenyx in the Past Three Years - All for Rare Diseases which Previously Had No A
Read more →RAREUltragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita (burosumab) for the Treatment of Tumor-Induced Osteomalacia (TIO) First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Sur
Ultragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita (burosumab) for the Treatment of Tumor-Induced Osteomalacia (TIO) First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Surgical Removal of Tumors TIO is Second FDA-Approved Indicat
Read more →RAREUltragenyx Announces Exercise of Option to Purchase Additional Stock of Arcturus Therapeutics
Ultragenyx Announces Exercise of Option to Purchase Additional Stock of Arcturus Therapeutics
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Ty
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) All Patients in Cohort 3 Demonstrate Increased Time to Hypoglycemia and a Substantial Reduct
Read more →RAREUltragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
Read more →RAREUltragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Tra
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency All three patients in Cohort 3 now confirmed responders All three complete
Read more →RAREUltragenyx to Present at BofA Securities Global Health Care Conference
Ultragenyx to Present at BofA Securities Global Health Care Conference
Read more →RAREUltragenyx Reports First Quarter 2020 Financial Results and Corporate Update
First quarter 2020 total revenue is $36.3 million First quarter 2020 Crysvita revenue in Ultragenyx territories is $28.8 million Maintains full year 2020 guidance based on COVID-19 impact to date NOVATO, Calif., May 06, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports First Quarter 2020 Financial Results and Corporate Update First quarter 2020 total revenue is $36.3 million First
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2020 Financial Results and Corporate Update First quarter 2020 total revenue is $36.3 million First quarter 2020 Crysvita revenue in Ultragenyx territories is $28.8 million Maintains full year 2020 guidan
Read more →RAREGeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome
GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2020 Financial Results and Corporate Update
NOVATO, Calif., April 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Wedn
Read more →RAREUltragenyx Announces Upcoming Data Presentations at American Society of Gene & Cell Therapy 2020 Virtual Annual Meeting
NOVATO, Calif., April 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that clinical, preclinical and manufa
Read more →RAREUltragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform Ultragenyx to receive $200
Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform Ultragenyx to receive $200 million upfront, including $125 million in cash and $75 m
Read more →RAREGeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome
GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome
Read more →RAREUltragenyx Announces Planned Transition of Chief Financial Officer
Ultragenyx Announces Planned Transition of Chief Financial Officer
Read more →RAREUltragenyx and Kyowa Kirin Announce FDA Acceptance and Priority Review Designation of Supplemental Biologics License Application for Crysvita (burosumab) for Tumor-Induced Osteomalacia (TIO)
NOVATO, Calif. and TOKYO, Feb. 27, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:
Read more →RAREUltragenyx to Present at SVB Leerink Global Healthcare Conference
Ultragenyx to Present at SVB Leerink Global Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update 2019 total revenue is $103.7 million; 201
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update 2019 total revenue is $103.7 million; 2019 Crysvita (burosumab) revenue to Ultragenyx is $87.3 million 2020 Crysvita revenue in Ultragenyx territo
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2019 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2019 Financial Results and Corporate Update
Read more →RAREGeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome
GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome
Read more →RAREUltragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita (burosumab) for Tumor-Induced Osteomalacia (TIO)
Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita (burosumab) for Tumor-Induced Osteomalacia (TIO)
Read more →RAREUltragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance
Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance Preliminary 2019 total revenue is approximate
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance Preliminary 2019 total revenue is approximately $102 million to $104 million Preliminary 2019 Crysvita revenue to Ultragenyx of approximately $86 mill
Read more →RAREUltragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency More uniform response in Cohort 3 with two confirme
Read more →RAREUltragenyx Announces Sale of Future European Royalties on Crysvita (burosumab) for $320 Million to Royalty Pharma
Ultragenyx Announces Sale of Future European Royalties on Crysvita (burosumab) for $320 Million to Royalty Pharma
Read more →RAREUltragenyx to Present at Piper Jaffray 31st Annual Healthcare Conference
Ultragenyx to Present at Piper Jaffray 31st Annual Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update Strong Crysvita (burosumab) Launch Continues with Approx
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update Strong Crysvita (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in the United States as of the end of the Third Qu
Read more →RAREUltragenyx Reports Third Quarter 2019 Financial Results and Corporate Update
Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2019 Financial Results and Corporate Update
NOVATO, Calif., Oct. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesd
Read more →RAREUltragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders
Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders
Read more →RAREUltragenyx and Kyowa Kirin Announce U.S. FDA Approves Label Update for Crysvita (burosumab) for the Treatment of X-Linked Hypophosphatemia (XLH)
Ultragenyx and Kyowa Kirin Announce U.S. FDA Approves Label Update for Crysvita (burosumab) for the Treatment of X-Linked Hypophosphatemia (XLH)
Read more →RAREUltragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita (burosumab) in Tumor-Induced Osteomalacia (TIO)
Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita (burosumab) in Tumor-Induced Osteomalacia (TIO)
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia Increased Time to
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic M
Read more →RAREUltragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Read more →RAREGeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102
GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102
Read more →RAREUltragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome Program aims to be first disease-modifying treatment for this serious neurogenetic disorder Ultragenyx receives an exclusive option
Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome Program aims to be first disease-modifying treatment for this serious neurogenetic disorder Ultragenyx receives an exclusive option to acquire GeneTx Novato, Calif. and Downers Grove, Ill.
Read more →RAREUltragenyx to Present at Wedbush PacGrow Healthcare Conference
Ultragenyx to Present at Wedbush PacGrow Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update Strong Crysvita (burosumab) Launch Continues with Appro
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update Strong Crysvita (burosumab) Launch Continues with Approximately 960 Patients on Reimbursed Commercial Therapy in the United States Ultragenyx Submits New Drug A
Read more →RAREUltragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders
Ultragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders
Read more →RAREUltragenyx Reports Second Quarter 2019 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2019 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Second Quarter 2019 Financial Results and Corporate Update
Read more →RAREUltragenyx and Arcturus Therapeutics Expand Existing Research Collaboration and License Agreement to Develop Additional Nucleic Acid Therapies for Rare Diseases • Scope of the collaboration expands to include up t
Ultragenyx and Arcturus Therapeutics Expand Existing Research Collaboration and License Agreement to Develop Additional Nucleic Acid Therapies for Rare Diseases NOVATO, Calif., and SAN DIEGO, Calif., June 19, 2019 - Ultragenyx Pharmaceutical, Inc. (Nasdaq: RARE), a biopharmaceut
Read more →RAREUltragenyx Expands Leadership Team and Promotes Erik Harris to Chief Commercial Officer
Ultragenyx Expands Leadership Team and Promotes Erik Harris to Chief Commercial Officer
Read more →RAREUltragenyx Announces Emil D. Kakkis, M.D., Ph.D. as Recipient of BIO's 2019 Henri A. Termeer Biotechnology Visionary Award
Ultragenyx Announces Emil D. Kakkis, M.D., Ph.D. as Recipient of BIO's 2019 Henri A. Termeer Biotechnology Visionary Award
Read more →RAREUltragenyx to Present at Bank of America Merrill Lynch Healthcare Conference
Ultragenyx to Present at Bank of America Merrill Lynch Healthcare Conference
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports First Quarter 2019 Financial Results and Corporate Update Strong Crysvita (burosumab) Launch Continues with Approx
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2019 Financial Results and Corporate Update Strong Crysvita (burosumab) Launch Continues with Approximately 730 Patients on Reimbursed Commercial Therapy in the United States NOVATO, Calif. - May 6, 2019 -
Read more →RAREUltragenyx Reports First Quarter 2019 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2019 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2019 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for First Quarter 2019 Financial Results and Corporate Update
Read more →RAREUltragenyx Announces UX007 Granted Fast Track Designation and Rare Pediatric Disease Designation by U.S. FDA for Treatment of Long-Chain Fatty Acid Oxidation Disorders
Ultragenyx Announces UX007 Granted Fast Track Designation and Rare Pediatric Disease Designation by U.S. FDA for Treatment of Long-Chain Fatty Acid Oxidation Disorders
Read more →RAREUltragenyx to Present Corporate Update at Analyst and Investor Day on April 17 in New York
Ultragenyx to Present Corporate Update at Analyst and Investor Day on April 17 in New York
Read more →RAREUltragenyx Announces Approval of Crysvita (burosumab) in Brazil for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children
Ultragenyx Announces Approval of Crysvita (burosumab) in Brazil for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children
Read more →RAREUltragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Ty
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia DTX401 Response in Time to Hypoglycemia and Improved Glucose Control Maintained or Improved in All T
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update Strong Launch Continues With More than 55
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update Strong Launch Continues With More than 550 Patients on Reimbursed Commercial Crysvita (burosumab) Therapy in the United States at End of 4th Quart
Read more →RAREUltragenyx and Kyowa Kirin Announce Positive 64-Week Results for Crysvita (burosumab) from Phase 3 Study in Children with X-linked Hypophosphatemia (XLH)
Ultragenyx and Kyowa Kirin Announce Positive 64-Week Results for Crysvita (burosumab) from Phase 3 Study in Children with X-linked Hypophosphatemia (XLH)
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2018 Financial Results and Corporate Update
NOVATO, Calif., Feb. 12, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesd
Read more →RAREUltragenyx Appoints Shehnaaz Suliman, M.D., to Board of Directors
Ultragenyx Appoints Shehnaaz Suliman, M.D., to Board of Directors
Read more →RAREUltragenyx Announces Positive Topline Data from Ongoing Long-Term Extension Study of UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders
Ultragenyx Announces Positive Topline Data from Ongoing Long-Term Extension Study of UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders
Read more →RAREUltragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storag
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia DTX401 Response Observed in All Three Patients, with Two Patients Demonstrating Clinical
Read more →RAREUltragenyx to Present at J.P. Morgan Healthcare Conference
Ultragenyx to Present at J.P. Morgan Healthcare Conference
Read more →RAREUltragenyx and Kyowa Kirin Announce Health Canada Approval of Crysvita (burosumab injection) for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children
Ultragenyx and Kyowa Kirin Announce Health Canada Approval of Crysvita (burosumab injection) for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children
Read more →RAREUltragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Mid-2019
Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Mid-2019
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Intent to Submit New Drug Application to U.S. FDA for UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Mid-2019 Novato, Calif. - November 14, 2018 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE),
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Third Quarter 2018 Financial Results and Corporate Update
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Third Quarter 2018 Financial Results and Corporate Update NOVATO, Calif. - November 5, 2018 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for se
Read more →RAREUltragenyx Reports Third Quarter 2018 Financial Results and Corporate Update
Ultragenyx Reports Third Quarter 2018 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2018 Financial Results and Corporate Update
NOVATO, Calif., Oct. 30, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Monda
Read more →RAREUltragenyx Announces Negative Topline Results from Phase 3 Study of UX007 in Patients with Glut1 DS with Disabling Movement Disorders
Ultragenyx Announces Negative Topline Results from Phase 3 Study of UX007 in Patients with Glut1 DS with Disabling Movement Disorders
Read more →RAREUltragenyx Announces Exclusive License to REGENXBIO AAV Vectors to Develop Gene Therapy for CDD (CDKL5 Deficiency Disorder)
Ultragenyx CEO to present Keynote Address at the 2018 CDKL5 Forum hosted by the Loulou Foundation NOVATO, Calif., Oct. 22, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious
Read more →RAREUltragenyx Announces Approval of Mepsevii (vestronidase alfa) in Brazil for the Treatment of Mucopolysaccharidosis VII
Ultragenyx Announces Approval of Mepsevii (vestronidase alfa) in Brazil for the Treatment of Mucopolysaccharidosis VII
Read more →RAREUltragenyx Announces Positive Topline Cohort 2 Results from Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose
Ultragenyx Announces Positive Topline Cohort 2 Results from Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose
Read more →RAREUltragenyx to Present at Jefferies Gene Therapy Summit
Ultragenyx to Present at Jefferies Gene Therapy Summit
Read more →RAREUltragenyx Expands Leadership Team and Appoints Dr. Wladimir Hogenhuis as Chief Operating Officer Novato, Calif.
Ultragenyx Expands Leadership Team and Appoints Dr. Wladimir Hogenhuis as Chief Operating Officer Novato, Calif. - September 17, 2018 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare
Read more →RAREUltragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders
Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders
Read more →RAREUltragenyx Announces Approval of Mepsevii (vestronidase alfa) in Europe for the Treatment of Mucopolysaccharidosis VII Mepsevii, an Enzyme Replacement Therapy, is the First Treatment Approved in the EU for Mucopolysaccha
Ultragenyx Announces Approval of Mepsevii (vestronidase alfa) in Europe for the Treatment of Mucopolysaccharidosis VII Mepsevii, an Enzyme Replacement Therapy, is the First Treatment Approved in the EU for Mucopolysaccharidosis VII Novato, Calif. - August 27, 2018 - Ultragenyx
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Second Quarter 2018 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2018 Financial Results and Corporate Update NOVATO, CA - August 2, 2018 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare an
Read more →RAREUltragenyx Second Quarter 2018 Financial Results Conference Call
Ultragenyx Second Quarter 2018 Financial Results Conference Call Attendees: Emil Kakkis, Shalini Sharp, Danielle Keatley Danielle Keatley - Opening Comments Good afternoon and welcome to the Ultragenyx Pharmaceutical financial results and corporate update conference call for t
Read more →RAREUltragenyx Reports Second Quarter 2018 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2018 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2018 Financial Results and Corporate Update
NOVATO, Calif., July 27, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, August 2, 20
Read more →RAREUltragenyx Announces First Patient Dosed in Phase 1/2 study of DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa); FDA Grants Fast Track Designation to Gene Therapy Program
Ultragenyx Announces First Patient Dosed in Phase 1/2 study of DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa); FDA Grants Fast Track Designation to Gene Therapy Program
Read more →RAREUltragenyx Announces Positive CHMP Opinion for Mepsevii™ (vestronidase alfa) For the Treatment of Mucopolysaccharidosis VII
NOVATO, Calif., June 29, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion recommend
Read more →RAREUltragenyx and Kyowa Kirin Announce Publication of Phase 2 Study Results Demonstrating that Crysvita (burosumab) Improved Outcomes in Children with X-linked Hypophosphatemia in the New England Journal of Medicine
Ultragenyx and Kyowa Kirin Announce Publication of Phase 2 Study Results Demonstrating that Crysvita (burosumab) Improved Outcomes in Children with X-linked Hypophosphatemia in the New England Journal of Medicine
Read more →RAREUltragenyx and Kyowa Kirin Announce Topline Phase 3 Study Results Demonstrating Superiority of Crysvita (burosumab) Treatment to Oral Phosphate and Active Vitamin D in Children with X-Linked Hypophosphatemia (XLH)
Ultragenyx and Kyowa Kirin Announce Topline Phase 3 Study Results Demonstrating Superiority of Crysvita (burosumab) Treatment to Oral Phosphate and Active Vitamin D in Children with X-Linked Hypophosphatemia (XLH)
Read more →RAREUltragenyx Reports First Quarter 2018 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2018 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports First Quarter 2018 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2018 Financial Results and Corporate Update NOVATO, CA - May 7, 2018 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ul
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2018 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for First Quarter 2018 Financial Results and Corporate Update
Read more →RAREUltragenyx and Kyowa Kirin Announce Crysvita (burosumab-twza) Now Launched in the U.S. for the Treatment of X-linked Hypophosphatemia (XLH) in Children and Adults
Ultragenyx and Kyowa Kirin Announce Crysvita (burosumab-twza) Now Launched in the U.S. for the Treatment of X-linked Hypophosphatemia (XLH) in Children and Adults
Read more →RAREUltragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia
Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia
Read more →RAREUltragenyx and Kyowa Kirin Announce FDA Approval of Crysvita (burosumab-twza) for the Treatment of Children and Adults with X-Linked Hypophosphatemia (XLH) First Approved Therapy for XLH in the U.S.; Only Treatment that
Ultragenyx and Kyowa Kirin Announce FDA Approval of Crysvita (burosumab-twza) for the Treatment of Children and Adults with X-Linked Hypophosphatemia (XLH) First Approved Therapy for XLH in the U.S.; Only Treatment that Targets the Underlying Cause of this Rare, Hereditary, Life
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigatio
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigational Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency Ureagenesis normalized in one patient and
Read more →RAREUltragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigational Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigational Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Read more →RAREKyowa Kirin and Ultragenyx Announce Crysvita (burosumab) Receives Conditional Marketing Authorization in Europe for the Treatment of X-Linked Hypophosphatemia in Children Crysvita is the First Treatment for XLH that Targ
Kyowa Kirin and Ultragenyx Announce Crysvita (burosumab) Receives Conditional Marketing Authorization in Europe for the Treatment of X-Linked Hypophosphatemia in Children Crysvita is the First Treatment for XLH that Targets the Underlying Cause of the Disease Crysvita Acknowle
Read more →RAREUNAUDITED PRO FORMA CONDENSED COMBINED FINANCIAL INFORMATION The following unaudited pro forma condensed combined financial information combines the historical consolidated financial information of Ultragenyx Pharmaceuti
UNAUDITED PRO FORMA CONDENSED COMBINED FINANCIAL INFORMATION The following unaudited pro forma condensed combined financial information combines the historical consolidated financial information of Ultragenyx Pharmaceutical Inc. (the Company or Ultragenyx) and Dimension Therapeu
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Fourth Quarter and Full Year 2017 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Fourth Quarter and Full Year 2017 Financial Results and Corporate Update NOVATO, CA - Feb. 20, 2018 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel product
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2017 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2017 Financial Results and Corporate Update
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full Year 2017 Financial Results and Corporate Update
NOVATO, Calif., Feb. 14, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Tuesday, February 20,
Read more →RAREUltragenyx to Present at Leerink Partners Global Healthcare Conference
NOVATO, Calif., Feb. 08, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, wi
Read more →RAREUltragenyx Appoints Camille L. Bedrosian, M.D. as Chief Medical Officer and Executive Vice President
Ultragenyx Appoints Camille L. Bedrosian, M.D. as Chief Medical Officer and Executive Vice President
Read more →RAREUltragenyx Announces Positive Interim Topline Results From First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Announces Positive Interim Topline Results From First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Read more →RAREUltragenyx Announces Update to UX007 Development Program in Long-Chain Fatty Acid Oxidation Disorder Patients
Ultragenyx Announces Update to UX007 Development Program in Long-Chain Fatty Acid Oxidation Disorder Patients
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Sells Priority Review Voucher for $130 million Novato, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Sells Priority Review Voucher for $130 million Novato, CA, - December 18, 2017 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare dis
Read more →RAREUltragenyx Sells Priority Review Voucher for $130 million
Ultragenyx Sells Priority Review Voucher for $130 million
Read more →RAREUltragenyx and Kyowa Kirin Announce Burosumab Receives Positive CHMP Opinion for the Treatment of X-Linked Hypophosphatemia in Children If approved, Burosumab would be the first targeted disease-modifying treatment for X
Ultragenyx and Kyowa Kirin Announce Burosumab Receives Positive CHMP Opinion for the Treatment of X-Linked Hypophosphatemia in Children If approved, Burosumab would be the first targeted disease-modifying treatment for XLH Tokyo, Japan, London, UK and Novato, CA- December 15, 2
Read more →RAREUltragenyx and Kyowa Kirin Announce Positive 48-Week Data from Adult Phase 3 Study of Burosumab (KRN23) in X-Linked Hypophosphatemia
Ultragenyx and Kyowa Kirin Announce Positive 48-Week Data from Adult Phase 3 Study of Burosumab (KRN23) in X-Linked Hypophosphatemia
Read more →RAREUltragenyx to Highlight Pipeline at Analyst and Investor Day on December 4
Ultragenyx to Highlight Pipeline at Analyst and Investor Day on December 4
Read more →RAREUltragenyx Announces Completion of Patient Dosing in First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Announces Completion of Patient Dosing in First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Announces FDA Approval of MEPSEVII (vestronidase alfa), the First Therapy for Progressive and Debilitating Rare Genetic Di
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces FDA Approval of MEPSEVII (vestronidase alfa), the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis VII Ultragenyx to Host Conference Call Today at 5:00pm ET Novato, CA, - Novem
Read more →RAREUltragenyx Announces FDA Approval of MEPSEVII (vestronidase alfa), the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis VII
Ultragenyx Announces FDA Approval of MEPSEVII (vestronidase alfa), the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis VII
Read more →RAREUltragenyx Completes Tender Offer and Acquisition of Dimension Therapeutics
Ultragenyx Completes Tender Offer and Acquisition of Dimension Therapeutics
Read more →RAREUltragenyx to Present at the Credit Suisse Healthcare Conference
NOVATO, Calif., Nov. 06, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executi
Read more →RAREUltragenyx Reports Third Quarter 2017 Financial Results and Corporate Update
Ultragenyx Reports Third Quarter 2017 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876 Ultragenyx Reports Third Quarter 2017 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Third Quarter 2017 Financial Results and Corporate Update NOVATO, CA - Nov 2, 2017 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ul
Read more →RAREUltragenyx and Kyowa Hakko Kirin Announce FDA Not Currently Planning to Hold Advisory Committee Meeting for Burosumab Biologics License Application
Ultragenyx and Kyowa Hakko Kirin Announce FDA Not Currently Planning to Hold Advisory Committee Meeting for Burosumab Biologics License Application
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2017 Financial Results and Corporate Update
NOVATO, Calif., Oct. 30, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, November 2,
Read more →RAREUltragenyx and Dimension Announce Expiration of Hart-Scott-Rodino Waiting Period for Ultragenyx’s Acquisition of Dimension
NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 26, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) (“Ultragenyx” or the “Company”), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Dimension Therapeuti
Read more →RAREUltragenyx and Kyowa Hakko Kirin Announce FDA Acceptance and Priority Review Designation of Burosumab’s Biologics License Application
NOVATO, Calif., TOKYO and LONDON, Oct. 10, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin) and Kyowa K
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 415-483-8257 Contact Kyowa Hakko Kirin Co. Ltd. Media Hiroki Nakamura +81-3-5205-7205 Email: media@kyowa-kirin.co.jp Ultragenyx and Kyowa Hakko Kirin
Contact Ultragenyx Pharmaceutical Inc. Contact Kyowa Hakko Kirin Co. Ltd. Email: media@kyowa-kirin.co.jp Ultragenyx and Kyowa Hakko Kirin Announce FDA Acceptance and Priority Review Designation of Burosumab s Biologics License Application Novato, CA, Tokyo, Japan and London,
Read more →RAREUltragenyx to Acquire Dimension Therapeutics
Ultragenyx to Acquire Dimension Therapeutics
Read more →RAREUltragenyx to Acquire Dimension Therapeutics Ultragenyx Enters into Definitive Agreement to Acquire Dimension for $6.00 Per Share in Cash in a Transaction Valued at Approximately $151 Million Based on Currently Outstandi
Ultragenyx to Acquire Dimension Therapeutics Ultragenyx Enters into Definitive Agreement to Acquire Dimension for $6.00 Per Share in Cash in a Transaction Valued at Approximately $151 Million Based on Currently Outstanding Shares Combined Company will be Strongly Positioned to
Read more →RAREUltragenyx Comments on Dimension Therapeutics Announcement that Ultragenyx s Offer is a Superior Proposal
Ultragenyx Comments on Dimension Therapeutics Announcement that Ultragenyx s Offer is a NOVATO, Calif., Oct. 02, 2017 (GLOBE NEWSWIRE) Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) ( Ultragenyx or the Company ), a biopharmaceutical company focused on the development of novel pro
Read more →RARERARE JPM 2017 Fall Biotech Conference Call Series
RARE JPM 2017 Fall Biotech Conference Call Series September 22, 2017 7:30 AM PST Conference Call Transcript Please disconnect now. At this time, all participants will be on a listen only mode for the entire duration of today s conference. I would now like to turn the call over
Read more →RAREUltragenyx Proposes to Acquire Dimension Therapeutics for $5.50 Per Share in Cash
Ultragenyx Proposes to Acquire Dimension Therapeutics for $5.50 Per Share in Cash September 18, 2017 8:30 AM PST Conference Call Transcript Ryan Martins, Vice President, Strategy and Introduction: Participants and Agenda Good afternoon and welcome to the Ultragenyx Pharmaceut
Read more →RAREUltragenyx and Kyowa Hakko Kirin Announce Additional Burosumab Data in X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO) at ASBMR
Ultragenyx and Kyowa Hakko Kirin Announce Additional Burosumab Data in X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO) at ASBMR
Read more →RAREUltragenyx to Present at the Morgan Stanley Global Healthcare Conference
Ultragenyx to Present at the Morgan Stanley Global Healthcare Conference
Read more →RAREUltragenyx to Present at the Baird 2017 Global Healthcare Conference
NOVATO, Calif., Aug. 31, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's Chief Executive
Read more →RAREUltragenyx Announces Burosumab Data Presentations at ASBMR 2017 Annual Meeting
NOVATO, Calif., Aug. 24, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced upcoming presentations of data highlighting burosumab for the
Read more →RAREUltragenyx and Kyowa Hakko Kirin Announce Submission of Burosumab's Biologics License Application in the US
Ultragenyx and Kyowa Hakko Kirin Announce Submission of Burosumab's Biologics License Application in the US
Read more →RAREUltragenyx Announces Top-Line Results from Phase 3 Study of Ace-ER in GNE Myopathy
Ultragenyx Announces Top-Line Results from Phase 3 Study of Ace-ER in GNE Myopathy
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 415-483-8257 Ultragenyx Announces Top-Line Results from Phase 3 Study of Ace-ER in GNE Myopathy Study did not meet its primary endpoint Novato, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Top-Line Results from Phase 3 Study of Ace-ER in GNE Myopathy Study did not meet its primary endpoint Novato, CA - August 22, 2017 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused o
Read more →RAREUltragenyx Reports Second Quarter 2017 Financial Results and Corporate Update
Ultragenyx Reports Second Quarter 2017 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 415-483-8257 Ultragenyx Reports Second Quarter 2017 Financial Results and Corporate Update Burosumab US BLA submission planned for August 2017 NOVATO,
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2017 Financial Results and Corporate Update Burosumab US BLA submission planned for August 2017 NOVATO, CA - July 27, 2017 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focu
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2017 Financial Results and Corporate Update
NOVATO, Calif., July 21, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, July 27, 201
Read more →RAREUltragenyx Provides Regulatory Update on Burosumab (KRN23)
NOVATO, Calif. , June 23, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has reached agreement with the FDA at a Pre-Biologic
Read more →RAREUltragenyx to Present at Goldman Sachs 38th Annual Global Healthcare Conference
NOVATO, Calif., June 07, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil Kakkis, M.D., Ph.D., the company's Chief Executive
Read more →RAREUltragenyx Announces Recombinant Human Beta-Glucuronidase Biologics License Application and Marketing Authorization Application Filed and Accepted for Review; FDA Grants Priority Review Status
Ultragenyx Announces Recombinant Human Beta-Glucuronidase Biologics License Application and Marketing Authorization Application Filed and Accepted for Review; FDA Grants Priority Review Status
Read more →RAREUltragenyx Reports First Quarter 2017 Financial Results and Corporate Update
Ultragenyx Reports First Quarter 2017 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports First Quarter 2017 Financial Results and Corporate Update Additional Phase 3 data on burosumab shows increased healing
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2017 Financial Results and Corporate Update Additional Phase 3 data on burosumab shows increased healing of fractures in adult XLH NOVATO, CA - May 4, 2017 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE),
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2017 Financial Results and Corporate Update
NOVATO, Calif., April 28, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, May 4, 2017
Read more →RAREUltragenyx Initiates Global Phase 3 Study of UX007 in Glut1 DS Patients with Disabling Movement Disorders
NOVATO, Calif., April 27, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the initiation of the Phase 3 study of UX007 for the treatme
Read more →RAREUltragenyx, Kyowa Hakko Kirin and Kyowa Kirin International Announce Positive 24-Week Data from Adult Phase 3 Study of Burosumab (KRN23) in X-Linked Hypophosphatemia
Ultragenyx, Kyowa Hakko Kirin and Kyowa Kirin International Announce Positive 24-Week Data from Adult Phase 3 Study of Burosumab (KRN23) in X-Linked Hypophosphatemia
Read more →RAREUltragenyx Appoints Deborah Dunsire, M.D., to Board of Directors
Ultragenyx Appoints Deborah Dunsire, M.D., to Board of Directors
Read more →RAREUltragenyx and Kyowa Kirin International Announce Positive Data from Pediatric Phase 2 Studies of Burosumab (KRN23) in X-Linked Hypophosphatemia
Ultragenyx and Kyowa Kirin International Announce Positive Data from Pediatric Phase 2 Studies of Burosumab (KRN23) in X-Linked Hypophosphatemia
Read more →RAREUltragenyx Announces Topline Data from Phase 2 UX007 Glucose Transporter Type-1 Deficiency Syndrome Seizure Study
Ultragenyx Announces Topline Data from Phase 2 UX007 Glucose Transporter Type-1 Deficiency Syndrome Seizure Study
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Update
Ultragenyx Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Update
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Update NOVATO, CA - February 16, 2017 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel pro
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full Year 2016 Financial Results and Corporate Update
NOVATO, Calif., Feb. 09, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, February 16,
Read more →RAREUltragenyx and Kyowa Kirin International Announce Marketing Authorization Application for KRN23 Filed and Accepted for Review by European Medicines Agency
NOVATO, Calif. and LONDON, Jan. 05, 2017 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and Kyowa Kirin International PLC (KKI), a wholly owned subsidiary of Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin), today announced that the European Medicines Agency (EMA
Read more →RAREUltragenyx Announces Positive Data from Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients
Ultragenyx Announces Positive Data from Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients
Read more →RAREUltragenyx Announces Withdrawal of Marketing Authorization Application for Aceneuramic Acid Prolonged Release (Ace-ER) in the European Union
Ultragenyx Announces Withdrawal of Marketing Authorization Application for Aceneuramic Acid Prolonged Release (Ace-ER) in the European Union
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Announces Withdrawal of Marketing Authorization Application for Aceneuramic Acid Prolonged Release (Ace-ER) in the European Un
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Withdrawal of Marketing Authorization Application for Aceneuramic Acid Prolonged Release (Ace-ER) in the European Union NOVATO, CA November 11, 2016 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
Read more →RAREUltragenyx Reports Third Quarter 2016 Financial Results and Corporate Update
NOVATO, Calif., Nov. 07, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter end
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports Third Quarter 2016 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Third Quarter 2016 Financial Results and Corporate Update NOVATO, CA November 7, 2016 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2016 Financial Results and Corporate Update
NOVATO, Calif., Oct. 31, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Monday, November 7, 20
Read more →RAREUltragenyx Announces First Patient Enrolled in Global Phase 3 Study of KRN23 in Pediatric Patients with X-Linked Hypophosphatemia (XLH)
NOVATO, Calif, Oct. 26, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the initiation of the Phase 3 study of KRN23 for the treatment
Read more →RAREUltragenyx to Present at Leerink Partners Roundtable Series
NOVATO, Calif., Sept. 27, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Execut
Read more →RAREUltragenyx Reports Positive Interim Data from Pediatric and Adult Phase 2 Studies of KRN23 in X-Linked Hypophosphatemia
Pediatric study demonstrates substantial reduction in bone disease and improvement in growth Adult study demonstrates increase in serum phosphorus and provides evidence of clinical improvement Company to host conference call today at 11am ET to discuss results NOVATO, Calif.,
Read more →RAREUltragenyx Reports Positive Interim Data from Phase 2 Study of KRN23 for the Treatment of Tumor-Induced Osteomalacia
Ultragenyx Reports Positive Interim Data from Phase 2 Study of KRN23 for the Treatment of Tumor-Induced Osteomalacia
Read more →RAREUltragenyx to Host Conference Call to Discuss KRN23 Data Presented at ASBMR 2016 Annual Meeting
NOVATO, Calif., Sept. 16, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Monday, September 19
Read more →RAREUltragenyx to Present at Baird 2016 Global Healthcare Conference
NOVATO, Calif., Sept. 01, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company’s Chief Financial Officer, w
Read more →RAREUltragenyx Announces KRN23 Data Presentations at ASBMR 2016 Annual Meeting
Ultragenyx Announces KRN23 Data Presentations at ASBMR 2016 Annual Meeting
Read more →RAREUltragenyx Reports Second Quarter 2016 Financial Results and Corporate Update
NOVATO, Calif., Aug. 08, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter end
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports Second Quarter 2016 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2016 Financial Results and Corporate Update NOVATO, CA August 8, 2016 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2016 Financial Results and Corporate Update
Ultragenyx to Host Conference Call for Second Quarter 2016 Financial Results and Corporate Update
Read more →RAREUltragenyx Announces Completion of Enrollment in Phase 3 Study of Aceneuramic Acid Extended Release (Ace-ER) in GNE Myopathy
Ultragenyx Announces Completion of Enrollment in Phase 3 Study of Aceneuramic Acid Extended Release (Ace-ER) in GNE Myopathy
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Announces Positive Topline Data from Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7 Stu
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Announces Positive Topline Data from Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7 endpoint of reduction in urinary GAG excretion and provides evidence of clinical improvement Company to
Read more →RAREUltragenyx Announces Positive Topline Data from Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7
Ultragenyx Announces Positive Topline Data from Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7
Read more →RAREUltragenyx to Present Phase 3 Data for Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7 (MPS 7) at 14th International Symposium on MPS and Related Diseases
Ultragenyx to Present Phase 3 Data for Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7 (MPS 7) at 14th International Symposium on MPS and Related Diseases
Read more →RAREUltragenyx Receives Breakthrough Therapy Designation for KRN23 in Pediatric Patients with X-Linked Hypophosphatemia
Ultragenyx Receives Breakthrough Therapy Designation for KRN23 in Pediatric Patients with X-Linked Hypophosphatemia
Read more →RAREUltragenyx Appoints Karah Parschauer as Executive Vice President, General Counsel
Ultragenyx Appoints Karah Parschauer as Executive Vice President, General Counsel
Read more →RAREUltragenyx and Takeda enter into a Collaboration to Develop and Commercialize Therapies for Rare Genetic Diseases
Ultragenyx and Takeda enter into a Collaboration to Develop and Commercialize Therapies for Rare Genetic Diseases
Read more →RAREUltragenyx Reports First Quarter 2016 Financial Results and Corporate Update
NOVATO, Calif., May 09, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter ende
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports First Quarter 2016 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2016 Financial Results and Corporate Update NOVATO, CA May 9, 2016 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultr
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2016 Financial Results and Corporate Update
NOVATO, Calif., April 29, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Monday, May 9, 2016 a
Read more →RAREUltragenyx and Saint Louis University's Center for World Health and Medicine Announce Research Collaboration to Advance Muscular Dystrophy Treatment
Ultragenyx and Saint Louis University's Center for World Health and Medicine Announce Research Collaboration to Advance Muscular Dystrophy Treatment
Read more →RAREUltragenyx Appoints Lars Ekman, M.D., Ph.D., to Board of Directors
NOVATO, Calif., March 21, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the appointment of Lars Ekman, M.D., Ph.D., to the company’s
Read more →RAREUltragenyx to Present at the Cowen and Company 36th Annual Health Care Conference
NOVATO, Calif., March 02, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company’s Chief Financial Officer, w
Read more →RAREUltragenyx Announces Data Presentations of rhGUS and rhPPCA at 12th Annual WORLD Symposium™
NOVATO, Calif., March 01, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced upcoming presentations of data highlighting recombinant huma
Read more →RAREUltragenyx Reports Fourth Quarter 2015 and Full-Year 2015 Financial Results and Corporate Update
NOVATO, Calif., Feb. 25, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the fourth quar
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports Fourth Quarter 2015 and Full-Year 2015 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Fourth Quarter 2015 and Full-Year 2015 Financial Results and Corporate Update NOVATO, CA February 25, 2016 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2015 Financial Results and Corporate Update
NOVATO, Calif., Feb. 18, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, February 25,
Read more →RAREUltragenyx to Present at the Leerink Partners Annual Healthcare Conference
NOVATO, Calif., Feb. 09, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company’s Chief Financial Officer, wi
Read more →RAREUltragenyx to Present at the J.P. Morgan Healthcare Conference
NOVATO, Calif., Jan. 05, 2016 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executi
Read more →RAREUltragenyx Announces First Patient Enrolled in Global Phase 3 Study of KRN23 in Adults With X-Linked Hypophosphatemia (XLH)
NOVATO, Calif., Dec. 03, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the initiation of the Phase 3 study of KRN23 for the treatmen
Read more →RAREUltragenyx Reports Positive Interim 40-Week Data From Pediatric Phase 2 Study of KRN23 in X-Linked Hypophosphatemia
Ultragenyx Reports Positive Interim 40-Week Data From Pediatric Phase 2 Study of KRN23 in X-Linked Hypophosphatemia
Read more →RAREUltragenyx to Present at the Oppenheimer Annual Healthcare Conference
NOVATO, Calif., Dec. 02, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executi
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 844-758-7273 Ultragenyx Reports Third Quarter 2015 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Third Quarter 2015 Financial Results and Corporate Update NOVATO, CA November 9, 2015 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and
Read more →RAREUltragenyx Reports Third Quarter 2015 Financial Results and Corporate Update
NOVATO, Calif., Nov. 09, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter end
Read more →RAREUltragenyx Announces Update to UX007 Development Program in Glucose Transporter Type-1 Deficiency Syndrome
NOVATO, Calif., Nov. 06, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced an update to its development plan for UX007 in Glut1 DS patie
Read more →RAREUltragenyx to Host Conference Call for Third Quarter 2015 Financial Results and Corporate Update
NOVATO, Calif., Nov. 03, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Monday, November 9, 20
Read more →RAREUltragenyx to Present at the Credit Suisse Annual Healthcare Conference
NOVATO, Calif., Nov. 03, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executi
Read more →RAREUltragenyx Pharmaceutical and Arcturus Therapeutics Announce a Research Collaboration and License Agreement to Develop RNA Therapeutics for Rare Diseases
Ultragenyx Pharmaceutical and Arcturus Therapeutics Announce a Research Collaboration and License Agreement to Develop RNA Therapeutics for Rare Diseases
Read more →RAREUltragenyx Announces Positive Interim Data From Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients
Ultragenyx Announces Positive Interim Data From Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients
Read more →RAREUltragenyx Announces Aceneuramic Acid Prolonged Release Marketing Authorization Application Filed and Accepted for Review by European Medicines Agency
Ultragenyx Announces Aceneuramic Acid Prolonged Release Marketing Authorization Application Filed and Accepted for Review by European Medicines Agency
Read more →RAREUltragenyx to Present at the Baird Health Care Conference
NOVATO, Calif., Sept. 2, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Sunil Agarwal, M.D., the company's Chief Medical Officer
Read more →RAREUltragenyx Announces Presentation of Data From Long-Chain Fatty Acid Oxidation Disorder Patients With Cardiomyopathy Treated With Triheptanoin
NOVATO, Calif., Sept. 1, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of open-label data from five infants with ca
Read more →RAREUltragenyx Reports Second Quarter 2015 Financial Results and Corporate Update
NOVATO, Calif., Aug. 13, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter end
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Robert Anstey 844-758-7273 Ultragenyx Reports Second Quarter 2015 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports Second Quarter 2015 Financial Results and Corporate Update NOVATO, CA August 13, 2015 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and
Read more →RAREUltragenyx to Host Conference Call for Second Quarter 2015 Financial Results and Corporate Update
NOVATO, Calif., Aug. 6, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Thursday, August 13, 20
Read more →RAREUltragenyx Announces Initiation of Phase 2 Study of Recombinant Human Beta-Glucuronidase in Patients Under Five Years Old With Mucopolysaccharidosis 7
Ultragenyx Announces Initiation of Phase 2 Study of Recombinant Human Beta-Glucuronidase in Patients Under Five Years Old With Mucopolysaccharidosis 7
Read more →RAREUltragenyx Announces Closing of Public Offering of Common Stock and Exercise of Underwriters' Option to Purchase Additional Shares
NOVATO, Calif., July 21, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the closing of its public offering of 2,530,000 shares of com
Read more →RAREUltragenyx Appoints John Pinion as Chief Quality Operations Officer and Senior Vice President, Analytical Sciences and Research
NOVATO, Calif., July 20, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has appointed John Pinion as Chief Quality Operations Offi
Read more →RAREUltragenyx Releases Positive Interim 40-Week Bone Treatment Data From Ongoing Pediatric Phase 2 Study of KRN23 in X-Linked Hypophosphatemia
NOVATO, Calif., July 9, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced positive interim data from the first 12 patients in the ongoin
Read more →RAREUltragenyx Names Daniel G. Welch as Chairman of the Board
NOVATO, Calif., June 25, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the Board of Directors has elected Daniel G. Welch as the Com
Read more →RAREUltragenyx Appoints Jayson Dallas, M.D., as Chief Commercial Officer and Senior Vice President
NOVATO, Calif., June 22, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has appointed Jayson Dallas, M.D., as its Chief Commercial
Read more →RAREUltragenyx to Present at the JMP Securities Healthcare Conference
Ultragenyx to Present at the JMP Securities Healthcare Conference
Read more →RAREUltragenyx Announces Completion of Enrollment in Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7
Ultragenyx Announces Completion of Enrollment in Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7
Read more →RAREUltragenyx Appoints Dennis Huang as Chief Technical Operations Officer and Senior Vice President
Ultragenyx Appoints Dennis Huang as Chief Technical Operations Officer and Senior Vice President
Read more →RAREUltragenyx Releases Positive 16-Week Interim Data From Pediatric Phase 2 Study of KRN23 in X-Linked Hypophosphatemia
Potential conditional approval pathway in Europe Phase 3 adult XLH program initiation expected in 2H 2015 NOVATO, Calif., June 2, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for
Read more →RAREUltragenyx Announces First Patient Enrolled in Global Phase 3 Study of Aceneuramic Acid (Sialic Acid) Extended Release in GNE Myopathy
NOVATO, Calif., May 28, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the initiation of the Phase 3 study of aceneuramic acid extend
Read more →RAREUltragenyx to Present at the Jefferies 2015 Global Healthcare Conference
NOVATO, Calif., May 26, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Sunil Agarwal, M.D., the company's Chief Medical Officer,
Read more →RAREUltragenyx Pharmaceutical Inc. Investors & Media Robert Anstey 844-758-7273 Ultragenyx Reports First Quarter 2015 Financial Results and Corporate Update NOVATO, CA
Contact Ultragenyx Pharmaceutical Inc. Ultragenyx Reports First Quarter 2015 Financial Results and Corporate Update NOVATO, CA May 11, 2015 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ult
Read more →RAREUltragenyx Reports First Quarter 2015 Financial Results and Corporate Update
NOVATO, Calif., May 11, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the first quarte
Read more →RAREUltragenyx to Host Conference Call for First Quarter 2015 Financial Results and Corporate Update
NOVATO, Calif., May 5, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Monday, May 11, 2015 at
Read more →RAREUltragenyx Granted Additional Orphan Drug Designations for Triheptanoin
NOVATO, Calif., April 23, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received two orphan drug designations for trihep
Read more →RAREUltragenyx Announces Positive Data From Investigator-Sponsored Trial of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome
Ultragenyx Announces Positive Data From Investigator-Sponsored Trial of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome
Read more →RAREUltragenyx Appoints Daniel G. Welch to Board of Directors
Ultragenyx Appoints Daniel G. Welch to Board of Directors
Read more →RAREUltragenyx Reports Fourth Quarter and Full-Year 2014 Financial Results and Corporate Update
NOVATO, Calif., March 25, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the fourth qua
Read more →RAREUltragenyx to Host Conference Call for Fourth Quarter and Full-Year 2014 Financial Results and Corporate Update
NOVATO, Calif., March 20, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it will host a conference call on Wednesday, March 25,
Read more →RAREUltragenyx Announces Presentation of Disease Burden Data in Adult Patients With X-Linked Hypophosphatemia
NOVATO, Calif., March 9, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of data highlighting the significant impairm
Read more →RAREUltragenyx to Present at the Barclays Global Healthcare Conference
Novato, CA, March 4, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Tom Kassberg, the company's Chief Business Officer, will
Read more →RAREUltragenyx to Present at the Cowen and Company 35th Annual Health Care Conference in Boston
Novato, CA, Feb. 25, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, wi
Read more →RAREUltragenyx Appoints Michael Narachi to Board of Directors
NOVATO, Calif., Feb. 24, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the appointment of Michael Narachi to the company's Board of
Read more →RAREUltragenyx Announces Full Exercise of Underwriters' Option to Purchase Additional Shares
Ultragenyx Announces Full Exercise of Underwriters' Option to Purchase Additional Shares
Read more →RAREUltragenyx Announces Positive 36-Week Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7
Ultragenyx Announces Positive 36-Week Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7
Read more →RAREUltragenyx Announces Intent to File for Conditional Approval in Europe for Sialic Acid Extended-Release Tablets in Hereditary Inclusion Body Myopathy
NOVATO, Calif., Jan. 12, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced its intent to file a Marketing Authorization Application (MAA
Read more →RAREUltragenyx Announces License of Intellectual Property Related to the Treatment of Huntington's Disease With Triheptanoin
Ultragenyx Announces License of Intellectual Property Related to the Treatment of Huntington's Disease With Triheptanoin
Read more →RAREUltragenyx Initiates New Development Program Studying KRN23 for the Treatment of Tumor-Induced Osteomalacia
NOVATO, Calif., Jan. 6, 2015 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the initiation of a new development program for the human monoclonal
Read more →RAREUltragenyx Announces Initiation of Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7
NOVATO, Calif., Dec. 15, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the dosing of the first patient in the pivotal Phase 3 study
Read more →RAREUltragenyx to Present at the Oppenheimer Healthcare Conference
Novato, CA, Dec. 3, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Tom Kassberg, the company's Chief Business Officer, will
Read more →RAREUltragenyx Reports Third Quarter 2014 Financial Results and Corporate Update
NOVATO, Calif., Nov. 10, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the third quart
Read more →RAREUltragenyx Granted EU Orphan Drug Designation for KRN23 for the Treatment of X-Linked Hypophosphatemia
Ultragenyx Granted EU Orphan Drug Designation for KRN23 for the Treatment of X-Linked Hypophosphatemia
Read more →RAREUltragenyx Granted Orphan Drug Designation for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome
Novato, CA, Oct. 27, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the FDA Office of Orphan Products Development has grante
Read more →RAREUltragenyx Announces Interim Data From Phase 2 Extension Study of Sialic Acid Extended-Release at International Congress of the World Muscle Society
NOVATO, Calif., Oct. 13, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of results from a Phase 2 extension study of
Read more →RAREUltragenyx to Present at the Leerink Partners Rare Disease Roundtable
Novato, CA, Sept. 24, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Execut
Read more →RAREUltragenyx Announces Positive Results From a Long-Term Phase 1/2 Study of KRN23 in Adult Patients With X-Linked Hypophosphatemia
NOVATO, Calif., Sept. 15, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of results from a long-term Phase 1/2 exten
Read more →RAREUltragenyx Announces Positive Interim Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7
NOVATO, Calif., Sept. 3, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of positive interim data from the Phase 1/2
Read more →RAREUltragenyx Reports Second Quarter 2014 Financial Results and Corporate Update
NOVATO, Calif., Aug. 11, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the second quar
Read more →RAREUltragenyx Announces License of Intellectual Property for the Treatment of Epilepsy and Other Seizure-Related Disorders With Triheptanoin
NOVATO, Calif., Aug. 5, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced a license agreement with UniQuest Pty Limited for intellectual
Read more →RAREUltragenyx Appoints Sunil Agarwal, M.D. as Chief Medical Officer and Senior Vice President
NOVATO, Calif., July 23, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has appointed Sunil Agarwal, M.D. as its Chief Medical Off
Read more →RAREUltragenyx Announces Initiation of a Phase 2 Study of KRN23 for Pediatric X-Linked Hypophosphatemia in the US and EU
NOVATO, Calif., July 1, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient screened and enrolled in the Phase 2 study of the
Read more →RAREUltragenyx Announces Results From Phase 1/2 Study of KRN23 in X-linked Hypophosphatemia in Adults
NOVATO, Calif., June 24, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of results from a multiple-dose study, condu
Read more →RAREUltragenyx to Present at the Goldman Sachs Global Healthcare Conference
Novato, CA, June 4, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executiv
Read more →RAREUltragenyx to Present at the Jefferies 2014 Global Healthcare Conference
Novato, CA, May 27, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, wil
Read more →RAREUltragenyx Announces KRN23 Phase 1/2 Study Data to be Presented at ICE/ENDO Meeting on June 23rd and June 24th
NOVATO, Calif., May 19, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the upcoming presentation of three abstracts from a Phase 1/2
Read more →RAREUltragenyx Reports First Quarter 2014 Financial Results and Corporate Update
NOVATO, Calif., May 12, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the first quarte
Read more →RAREUltragenyx Announces Positive Data From Phase 2 Study of Sialic Acid Extended-Release at Emerging Sciences Session of American Academy of Neurology Annual Meeting
NOVATO, Calif., April 30, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of detailed results from a 48-week Phase 2
Read more →RAREUltragenyx Announces Preliminary Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7
NOVATO, Calif., March 27, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of preliminary data from the Phase 1/2 stud
Read more →RAREUltragenyx Reports Fourth Quarter and Full Year 2013 Financial Results
NOVATO, Calif., March 24, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and business highlights for the fourth
Read more →RAREUltragenyx to Present at the BioCentury Future Leaders in the Biotech Industry Conference
Novato, CA, March 21, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Execut
Read more →RAREUltragenyx Announces First Patient Enrolled in Phase 2 Study of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome
Novato, CA, March 11, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient enrolled in the Phase 2 study of triheptanoin (U
Read more →RAREUltragenyx to Present at Cowen and Company 34th Annual Health Care Conference in Boston
Novato, CA, Feb. 26, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, wi
Read more →RAREUltragenyx Announces Presentation of Data From a Single Patient Treated With Recombinant Human Beta-Glucuronidase at 10th Annual World Lysosomal Disease Network Symposium
NOVATO, Calif., Feb. 12, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced data presentations from a single patient treated with recombi
Read more →RAREUltragenyx Announces Initiation of Phase 2 Study for Patients with Long-Chain Fatty Acid Oxidation Disorders
NOVATO, Calif., Feb. 11, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the first patient has been enrolled in an open-label Pha
Read more →RAREUltragenyx Appoints Clay Siegall, Ph.D. and Matthew Fust to Board of Directors
NOVATO, Calif., Feb. 6, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the appointment of Clay B. Siegall, Ph.D. and Matthew Fust to
Read more →RAREUltragenyx Announces Closing of Initial Public Offering and Exercise of Underwriters' Option to Purchase Additional Shares
NOVATO, Calif., Feb. 5, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the closing of its initial public offering of 6,624,423 shares of common sto
Read more →RAREUltragenyx Announces Pricing of Initial Public Offering
NOVATO, Calif., Jan. 30, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the pricing of its initial public offering of 5,760,369 shares of its commo
Read more →RAREUltragenyx Announces Results From Phase 2 Study of Sialic Acid Extended-Release Treatment in Hereditary Inclusion Body Myopathy
NOVATO, Calif., Dec. 20, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline results from a 48-week Phase 2 clinical study of sialic acid extend
Read more →RAREUltragenyx Investigational New Drug Application for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome is in Effect
Ultragenyx Investigational New Drug Application for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome is in Effect
Read more →RAREUltragenyx Announces Initiation of Phase 1/2 Study of Recombinant Human Beta-Glucuronidase (rhGUS) in Mucopolysaccharidosis Type 7 (MPS 7)
Ultragenyx Announces Initiation of Phase 1/2 Study of Recombinant Human Beta-Glucuronidase (rhGUS) in Mucopolysaccharidosis Type 7 (MPS 7)
Read more →RAREUltragenyx Announces the Presentation of Data from a Single Dose Phase 1 Study, Conducted by Kyowa Hakko Kirin Co. Ltd. (KHK), of KRN23 in X-linked Hypophosphatemia (XLH) in Adults
Ultragenyx Announces the Presentation of Data from a Single Dose Phase 1 Study, Conducted by Kyowa Hakko Kirin Co. Ltd. (KHK), of KRN23 in X-linked Hypophosphatemia (XLH) in Adults
Read more →RAREUltragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress
Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 18th Annual World Muscle Society Congress
Read more →RAREUltragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Ultragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Read more →RAREUltragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia
Ultragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia
Read more →RAREUltragenyx Announces Presentation of Three Abstracts at the 12th International Congress of Inborn Errors of Metabolism (ICIEM 2013)
Ultragenyx Announces Presentation of Three Abstracts at the 12th International Congress of Inborn Errors of Metabolism (ICIEM 2013)
Read more →RAREUltragenyx Investigational New Drug Application for UX007 is Active
Ultragenyx Investigational New Drug Application for UX007 is Active
Read more →RAREUltragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial Testing UX003 in Mucopolysaccharidosis Type 7 (MPS 7)
Ultragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial Testing UX003 in Mucopolysaccharidosis Type 7 (MPS 7)
Read more →RAREUltragenyx to Present at Upcoming Healthcare Investor Conferences
Ultragenyx to Present at Upcoming Healthcare Investor Conferences
Read more →RAREUltragenyx Initiates New Development Program Studying Triheptanoin (UX007) for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Ultragenyx Initiates New Development Program Studying Triheptanoin (UX007) for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Read more →RAREUltragenyx Gains Worldwide Rights to Triheptanoin (UX007)
Ultragenyx Gains Worldwide Rights to Triheptanoin (UX007)
Read more →RAREUltragenyx Gains Worldwide Rights for Triheptanoin (UX007)
NOVATO, Calif., July 11, 2013 (GLOBE NEWSWIRE) -- Ultragenyx announced that it has expanded its exclusive license from Baylor Research Institute (BRI) in Dallas, Texas, to develop and commercialize triheptanoin outside of North America. The global license includes rights to paten
Read more →RAREUltragenyx Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
Ultragenyx Announces a Positive Signal in Interim Data from Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
Read more →RAREUltragenyx to Present at the Jefferies 2013 Global Healthcare Conference
NOVATO, Calif., May 29, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company, today announced its participation as a presenting company at the Jefferies 2013 Global Healthcare Conference on June 3, 2013. Emil D. Kakkis, M.D., Ph.D., the company's Chief
Read more →RAREUltragenyx Advances Clinical Development of UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Ultragenyx Advances Clinical Development of UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Read more →RAREUltragenyx Appoints Eric Yuen, MD as Chief Medical Officer and Senior Vice President
NOVATO, Calif., May 1, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced it has appointed Eric Yuen, MD as its Chief Medical Officer and Senior Vice
Read more →RAREUltragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy
Ultragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy
Read more →RAREUltragenyx to Present at the 20th Annual Future Leaders in the Biotech Industry Conference
Ultragenyx to Present at the 20th Annual Future Leaders in the Biotech Industry Conference
Read more →RAREUltragenyx to Present at the Cowen and Company 33rd Annual Health Care Conference
Ultragenyx to Present at the Cowen and Company 33rd Annual Health Care Conference
Read more →RAREUltragenyx Announces In-Licensing of Clinical-Stage Product Triheptanoin
Ultragenyx Announces In-Licensing of Clinical-Stage Product Triheptanoin
Read more →RAREUltragenyx to Present at the 2013 31st Annual J.P. Morgan Healthcare Conference
Ultragenyx to Present at the 2013 31st Annual J.P. Morgan Healthcare Conference
Read more →RAREUltragenyx Raises $75 Million in Oversubscribed Crossover Financing
Ultragenyx Raises $75 Million in Oversubscribed Crossover Financing
Read more →RAREUltragenyx Announces Completion of Phase 2 Study Enrollment for UX001 in Hereditary Inclusion Body Myopathy
Ultragenyx Announces Completion of Phase 2 Study Enrollment for UX001 in Hereditary Inclusion Body Myopathy
Read more →RAREUltragenyx Announces Three Abstracts Accepted for Poster Presentation at 17th Annual World Muscle Society Congress
Ultragenyx Announces Three Abstracts Accepted for Poster Presentation at 17th Annual World Muscle Society Congress
Read more →RAREUltragenyx to Present at the 11th Annual BIO Investor Forum in San Francisco
Ultragenyx to Present at the 11th Annual BIO Investor Forum in San Francisco
Read more →RAREUltragenyx to Present at the Stifel Nicolaus Weisel Healthcare Conference 2012 in Boston
Ultragenyx to Present at the Stifel Nicolaus Weisel Healthcare Conference 2012 in Boston
Read more →RAREUltragenyx to Present at the Canaccord Genuity 32nd Annual Growth Conference on August 16, 2012
Ultragenyx to Present at the Canaccord Genuity 32nd Annual Growth Conference on August 16, 2012
Read more →RAREUltragenyx to Present at the 2012 Wedbush PacGrow Life Sciences Management Access Conference on August 15
Ultragenyx to Present at the 2012 Wedbush PacGrow Life Sciences Management Access Conference on August 15
Read more →RAREUltragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy, a Rare Neuromuscular Disease
Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy, a Rare Neuromuscular Disease
Read more →RAREUltragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Read more →RAREUltragenyx Relocates Company Headquarters to Support Operational Expansion
Ultragenyx Relocates Company Headquarters to Support Operational Expansion
Read more →RAREUltragenyx Appoints Shalini Sharp as Chief Financial Officer and Senior Vice President, Finance
Ultragenyx Appoints Shalini Sharp as Chief Financial Officer and Senior Vice President, Finance
Read more →RAREUltragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7
Ultragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7
Read more →RAREUltragenyx Announces the Completion of the Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Ultragenyx Announces the Completion of the Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Read more →RAREUltragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 (MPS 7)
Read more →RAREUltragenyx In-Licenses Therapeutic Program for Rare Genetic Disease Mucopolysaccharidosis Type 7 from St. Louis University
Ultragenyx In-Licenses Therapeutic Program for Rare Genetic Disease Mucopolysaccharidosis Type 7 from St. Louis University
Read more →RAREUltragenyx Appoints Tom Kassberg as Chief Business Officer and Announces Management Team
Ultragenyx Appoints Tom Kassberg as Chief Business Officer and Announces Management Team
Read more →RAREUltragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
Read more →RAREUltragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
Ultragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
Read more →RAREUltragenyx Announces Series A Financing
Ultragenyx Announces Series A Financing
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