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=