Sarepta Therapeutics Shares Clinical Data and Integrated Analysis for SRP-9001, its investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

Sarepta has just presented new results and analyses that demonstrate a consistent, and statistically significant functional benefit of their gene-therapy product SRP-9001 in Duchenne patients versus a propensity-weighted external natural history control. 

The new data from studies 101, 102 and 103 demonstrated consistently meaningful results and importantly demonstrated sustained functional benefit over the 2- and 4-year timeframe.  

The safety profile was consistent with that previously reported; there was one incidence of myocarditis that resolved after treatment and no clinically relevant complement activation was observed. 

The full company press release can be found here.