Full Press Release Details
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 8 - 11, 2026, in Orlando, Florida.
At MDA 2026, Sarepta will present new and ongoing evidence across its rare neuromuscular portfolio, including commercially available gene therapy and exon-skipping treatments in Duchenne muscular dystrophy. Presentations include a late-breaking oral presentation on delandistrogene moxeparvovec gene therapy from the Phase 3 EMBARK study (Part 1) up to three years post-infusion compared with a matched external control. Another abstract will feature caregiver-reported impressions from the Phase 3 EMBARK study through two years of follow-up, offering complementary perspectives on treatment impact beyond clinician-reported and performance-based outcomes. Additionally, a safety analysis of several delandistrogene moxeparvovec clinical studies with up to 7.5 years of patient follow-up will be presented; pooled data will include treatment-related adverse events that most commonly occurred within the first 60 days post-infusion.
Sarepta will also present data from across its exon skipping franchise, including Phase 3 results from ESSENCE for golodirsen and casimersen. Also, a new real-world analysis that explores survival in patients treated with exon skipping medicines will be presented, as well as interim real-world findings from the Phase 4 EVOLVE study describing long-term safety and loss of ambulation as observed in clinical practice.
“At MDA we’re sharing data that reinforce dystrophin restoration as a foundational therapy and its ability to slow Duchenne disease progression over time,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. “We want to bring forward a data-driven view of treatment experience in clinical and real-world settings, including longer-term functional outcomes, pooled safety learnings, and caregiver-reporter perspectives that provide complementary insights into treatment effect in a subset of patients treated in EMBARK. Our goal is to ensure clinicians and families have the information they need to make treatment decisions with confidence.”
Sarepta Podium Presentation:
| 480LB: Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: EMBARK Functional Outcomes and Safety up to 3 Years Post-Infusion(late-breaker) | Poster:March 1010:30 a.m. – 1:30 p.m. ET4 – 4:30 p.m. ET6 – 8 p.m. ETOral:March 112:30 – 2:45 p.m. ET |
Sarepta Poster Presentations(*Denotes encore presentation):
| 22 S: Efficacy and Safety of Golodirsen and Casimersen Compared with Placebo in Duchenne Muscular Dystrophy (ESSENCE): Phase 3 Topline Results | March 86 – 8 p.m. ET | |
| 25 S: 2025 Interim Analysis of EVOLVE: A Long-Term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice | March 86 – 8 p.m. ET | |
| 71 S: A Real-World Target Trial Emulation of Eteplirsen, Golodirsen, and Casimersen to Evaluate Survival Among Patients with Duchenne Muscular Dystrophy | March 86 – 8 p.m. ET | |
| 77 S: Caregiver Global Impressions of Delandistrogene Moxeparvovec in Patients with Duchenne Muscular Dystrophy: Findings from EMBARK 2-Year Follow-Up | March 86 – 8 p.m. ET | |
| 56 S: Model-Based Evaluation of Delandistrogene Moxeparvovec Adeno-Associated Virus Pharmacokinetics and Safety Implications* | March 86 – 8 p.m. ET | |
| 478LB: Pooled Safety Analysis from Phase 1 to Phase 3 Clinical Trials of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy(late-breaker) | March 1010:30 – 1:30 p.m. ET4 – 4:30 p.m. ET6 – 8 p.m. ET | |
| 272 T: Quantitation of Dystrophin Expression in Patients with Duchenne Muscular Dystrophy by Western Blot Analysis Adjusted for Muscle Content | March 10Poster Reception:10:30 – 1:30 p.m. ET4 – 4:30 p.m. ET6 – 8 p.m. ET | |
| 319 T: Expression of SGCA and Safety Following Treatment with Patidistrogene Bexoparvovec in Patients with LGMD2D/R3: Results from a Phase 1b Study | March 10Poster Reception:10:30 – 1:30 p.m. ET4 – 4:30 p.m. ET6 – 8 p.m. ET |
The full MDA 2026 program is available here:https://www.mdaconference.org. Sarepta abstracts and presentations will be available on Sarepta.com in theEvents & Presentationssection following the MDA embargo.
About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visitwww.sarepta.comor follow us onLinkedIn,X,InstagramandFacebook.
Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Forward-Looking StatementsThis press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements related to our research and development programs, clinical trials, technologies, scientific approaches, products and product candidates; and expected plans and milestones, including presenting certain data and findings at MDA.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; we may not be able to execute on our business plans, including meeting expected or planned regulatory milestones and timelines, clinical development plans, and bringing products to markets for various reasons including possible limitations of financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.