Sarepta Therapeutics to Present New Data from its Neuromuscular Portfolio at 2024 World Muscle Society Congress
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
Podium Presentation:
19O: Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 |
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Late Breaking Poster:
726LBP: Long-term safety and tolerability of delandistrogene moxeparvovec in Duchenne muscular dystrophy: phase 1 to phase 3 clinical trials |
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Poster Presentations (*Denotes encore presentation)
391P: Agreement and accuracy of ambulatory definitions in Duchenne muscular dystrophy (DMD): a cross-sectional analysis* |
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94P: JOURNEY: a natural history study of Limb Girdle muscular dystrophies R3-R5: baseline characteristics of study cohort |
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423P: Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy: Phase 3 EMBARK primary results* |
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424P: Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: Phase 1B trial (ENDEAVOR) |
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425P: Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne Muscular dystrophy (DMD): a phase 1/2a study |
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428P: Cardiac MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 |
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The full WMS 2024 program is available at https://www.wms2024.com/page/programme.
About
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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