29.01.2024 13:56:27
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Sarepta Reports Positive Data From MOMENTUM Study Of SRP-5051 In Duchenne Muscular Dystrophy
(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday announced positive data from Part B of Phase 2 MOMENTUM study of SRP-5051 for the treatment of patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
The study was designed to assess dystrophin protein levels in skeletal muscle tissue following SRP-5051 treatment. Data from the study showed that at the target dose, SRP-5051 resulted in mean dystrophin expression of 5.17%, and mean exon skipping of 11.11% at 28 weeks.
"The data suggest a favorable benefit-risk profile for SRP-5051 and we look forward to discussing the results and next steps with FDA," ," said Louise Rodino-Klapac, executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics.
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