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Sarepta Therapeutics, Inc.
Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies
Jul 6 2022
4 min read

Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies

  • Sarepta and its partner Roche present new results and analyses at the International Congress on Neuromuscular Diseases (ICNMD), which demonstrate that SRP-9001 shows consistent, statistically significant functional benefits in individuals with Duchenne versus a propensity-weighted external control that continue to positively diverge from natural history disease course
  • In the 20-patient Cohort 1 of SRP-9001-103 (ENDEAVOR), SRP-9001-treated participants improved 4 points from their pre-therapy baselines, and 3.8 points (unadjusted means) and 3.2 points (least squared means) at 52 weeks on the North Star Ambulatory Assessment (NSAA) compared to a propensity-weighted external control group (p=