Full Press Release Details
SAN RAFAEL, Calif. , Sept. 5, 2017 /PRNewswire/ -- BioMarin (Nasdaq: BMRN ) announced that the company will present data in three platform presentations and fifteen poster presentations at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) being held September 5-8, 2017 in Rio de Janeiro, Brazil . Presentations span a range of areas in rare genetic diseases, including neuronal ceroid lipofuscinosis type 2 (CLN2) disease, phenylketonuria (PKU) and mucopolysacchardosis (MPS).
Platform presentations include preliminary safety and pharmacodynamic response data from a Phase 1/2 study of ICV BMN 250, a novel enzyme replacement therapy for the treatment of Sanfilippo B (MPS IIIB), a long-term safety and efficacy analysis of cerliponase alfa to treat children with CLN2 disease, as well as updated five-year safety and efficacy data evaluating pegvaliase to treat adults with PKU.
Listing of Posters and Presentations Related to BioMarin Products and Programs at the 13th International Congress of Inborn Errors of Metabolism (All times are local)
Platform Presentations
| Title | Authors |
| Preliminary safety and pharmacodynamics response data from a phase 1/2 study of ICV BMN 250, a novel enzyme replacement therapy for the treatment of Sanfilippo syndrome type B (MPS IIIB) Parallel Session 1: Wednesday, September 6 from 12:00-12:15 | Steve Maricich, MD, PhD, BioMarin Pharmaceutical Inc. Novato, CA, USA |
| Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter extension study Parallel Session 1: Wednesday, September 6 from 12:00-12:15 | Marina Trivisano, MD, Bambino Gesú Children's Hospital, IRCCS, Rome, Italy |
| Phase 2 long-term pegvaliase treatment for adults with phenylketonuria: updated year 5 safety and efficacy data from the PAL-003 extension Parallel Session 7: Wednesday, September 6 from 14:45-15:00 | Jerry Vockley, MD, PhD, Children's Hospital of Pittsburgh and University of Pittsburgh, Pittsburgh, PA, USA |
Poster Presentations
| MPS | |
| Title | Authors |
| Design and rationale of ongoing observational and treatment studies for BMN 250, a novel enzyme replacement therapy for Sanfilippo syndrome type B (MPS IIIB) Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 597 | Shaywitz A, Maricich S, Yu H, Kent S |
| Presenting signs and symptoms of MPS: results of an international physician survey Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 649 | Clarke L, Ellaway C, Foster H, Giugliani R, Goizet C, Goring S, Hawley S, Jurecki E, Khan Z, Lampe C, Martin K, McMullen S, Mitchell J, Mubarack F, Muenzer J, Sivri S, Stewart F, Tylki-Szymanska A, White K, Wijburg F |
| Presenting signs and symptoms of MPS: results of systematic literature analysis Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 662 | Mubarack F, Clarke L, Ellaway C, Foster H, Giugliani R, Goizet C, Goring S, Hawley S, Jurecki E, Khan Z, Lampe C, Martin K, McMullen S, Mitchell J, Muenzer J, Sivri S, Stewart FJ, Tylki-Szymanska A, White K, Wijburg F |
| Mucopolysaccharidosis type VI (MPS VI) and molecular analysis: a review of published classified variants in the ARSB gene Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 674 | Tomanin R, Karageorgos L, AlSayed M, Bailey M, Izzo E, Miller N, Sakuraba H, Zanetti A, Hopwood JJ |
| Impact of elosulfase alfa treatment on patient-reported outcomes in Morquio A syndrome: results from the first year of an English managed access agreement Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 807 | Lavery C, Jones SA, Hughes D, Murphy E, Jovanovic A, Hendriksz C, Cleary M, Hiwot T, Vijay S |
| CLN2 | |
| Title | Authors |
| Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter extension study Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 787 | Trivisano M, Schulz A, Specchio N, Gissen P, de los Reyes E, Cahan H, Slasor P, Ajayi T, Jacoby D |
| PKU | |
| Title | Authors |
| Phase 3 PRISM-2 long-term extension study evaluating efficacy and safety of pegvaliase for treatment of adults with phenylketonuria Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 285 | Vockley J, Levy H, Amato S, Zori R, Thomas J, Burton B, Harding C, Longo N, Posner J, Bilder D, Olbertz J, Gu Z, Lau K, Lin M, Larimore K, Dimmock D |
| Phase 2 long-term pegvaliase treatment for adults with phenylketonuria: updated year 5 safety and efficacy data from the PAL-003 extension Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 288 | Zori R, Levy H, Longo N, Lau K, Rosen O, Li M, Larimore K, Decker C, Weng H, Vockley J |
| Seventh interim analysis of the Kuvan ® Adult Maternal Paediatric European Registry (KAMPER): interim results in BH4 deficiency patients Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 295 | Van Spronsen FJ, Burlina A, Alm J, Belanger-Quintana A, Feillet F, Lagler FB, Muntau AC, Trefz FK, Jurecki ER, Kittus R, Alvarez I |
| Seventh interim analysis of the Kuvan ® Adult Maternal Paediatric European Registry (KAMPER): interim results in phenylketonuria patients Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 296 | Van Spronsen FJ, Muntau AC, Lagler FB, Feillet F, Alm J, Burlina A, Belanger-Quintana A, Alvarez I, Lilienstein J, Jurecki E, Trefz FK |
| Interim analysis of the phenylketonuria (PKU) patients enrolled in the PKUDOS registry Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 297 | Lilienstein J, Burton B, Grant M, Ficicioglu C, Kopesky J, Nguyen-Driver MD, Moore C, Jurecki E, Longo N |
| An interim analysis of the Kuvan ® Adult Maternal Paediatric European Registry (KAMPER) and phenylketonuria developmental outcomes and safety (PKUDOS) registries: pregnancies Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 298 | Feillet F, Ficicioglu C, Lagler FB, Longo N, Alm J, Muntau AC, Burlina A, Belnager-Quintana A, Trefz FK, Kittus R, Jurecki E, Alvarez I, Lilienstein J, Burton B |
| Estimation of resource use and quality of life in phenylketonuria (PKU) patients in Ireland Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 262 | Pastores G, Bracken J, Hughes J, Rogers Y, Stenson C, Clark A, Lloyd A, Alvarez I, Jain M, Kha A, Monavari A |
| Short-term biological variance of PHE in patients with phenylketonuria Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 250 | Potter M, Pendar A, Langley E, Geraghty M |
| Amino acid level correlations between tandem mass spectrometry and ultra-performance liquid chromatography and the clinical relevance for phenylketonuria management Presentation: Wednesday, September 6 from 17:30-20:00 Poster/Presentation: 251 | Potter M, Pendar A, Langley E, Geraghty M |
Links to Full Prescribing Information on BioMarin Products
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.
For additional information, please visit www.BMRN.com . Information on BioMarin's website is not incorporated by reference into this press release.
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| Traci McCarty | Debra Charlesworth |
| BioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. |
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