Sarepta Therapeutics reported positive early-stage clinical results for its next generation of muscular dystrophy treatments, including RNAi drugs developed with partner Arrowhead Pharmaceuticals. The company said the medicines appear to be safe and effective in treating muscle-weakening conditions.
The long-awaited results come from Phase 1/2 studies evaluating the therapies' safety profile and initial efficacy signals in patients with muscular dystrophy. According to Sarepta, the data suggest potential for the two investigational medicines to address these rare muscle-weakening disorders.