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Sarepta Therapeutics to Present New Data from its Neuromuscular Portfolio at 2024 World Muscle Society Congress
09/26/24 8:30 AM EDT
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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iestepan@sarepta.com
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tsorrentino@sarepta.com
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