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Shares of Sarepta Therapeutics (SRPT) jumped 6% in pre-market trading on Monday after the company announced positive three-year topline results from its Phase 3 EMBARK study evaluating ELEVIDYS, a gene therapy for ambulatory patients with Duchenne Muscular Dystrophy (DMD).
The data showed that patients treated with ELEVIDYS experienced a significant and durable slowing of disease progression compared with an external control group. Three years after treatment, patients maintained North Star Ambulatory Assessment (NSAA) scores above baseline, while untreated patients showed the expected decline.
ELEVIDYS reduced the rate of functional decline by about 70% or more on key measures, including Time to Rise and the 10-meter walk/run, with the treatment effect increasing over time. No new safety concerns were identified, supporting ELEVIDYS’ manageable safety profile.
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