CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the 29th Annual Congress of the World Muscle Society 2024 Congress (WMS 2024), taking place Oct. 8-12, 2024, in Prague, Czechia.
Among the data to be presented are new safety and efficacy results from several studies in the delandistrogene moxeparvovec clinical development program, including data from Studies 9001-101, 9001-103 (ENDEAVOR) and 9001-301 (EMBARK).
“New data to be presented at WMS from clinical studies of delandistrogene moxeparvovec include the first look at skeletal muscle and cardiac MRI outcomes from the EMBARK study, a late-breaking analysis of safety data from across all delandistrogene moxeparvovec clinical studies, and five-year functional results from Study SRP-9001-101, the longest-term data to date for a gene therapy in Duchenne,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. “The results add to the substantial and growing body of data for delandistrogene moxeparvovec.”
Podium Presentation:
19O: Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 | Oct. 12, 2024 1:45-2:00 a.m. EDT (07:45-08:00 CEST) |
Late Breaking Poster:
726LBP: Long-term safety and tolerability of delandistrogene moxeparvovec in Duchenne muscular dystrophy: phase 1 to phase 3 clinical trials | Oct. 11, 2024 9:45-10:45 a.m. EDT (15:45-16:45 CEST) |
Poster Presentations (*Denotes encore presentation)
391P: Agreement and accuracy of ambulatory definitions in Duchenne muscular dystrophy (DMD): a cross-sectional analysis* | Oct. 9, 2024 8:30-9:30 a.m. EDT (14:30-15:30 CEST) |
94P: JOURNEY: a natural history study of Limb Girdle muscular dystrophies R3-R5: baseline characteristics of study cohort | Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
423P: Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy: Phase 3 EMBARK primary results* | Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
424P: Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: Phase 1B trial (ENDEAVOR) | Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
425P: Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne Muscular dystrophy (DMD): a phase 1/2a study | Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
428P: Cardiac MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 | Oct. 9, 2024 11:15 a.m.-12:15 p.m. EDT (17:15-18:15 CEST) |
The full WMS 2024 program is available at https://www.wms2024.com/page/programme.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X (formerly Twitter), Instagram and Facebook.
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Forward-Looking Statements
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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.
Contacts
Investor Contact:
Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media Contact:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
