CAMBRIDGE, Mass., September 26, 2024--(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
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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