Sarepta Therapeutics reports that its gene therapy Elevidys for Duchenne muscular dystrophy shows continued benefits three years post-treatment, despite facing regulatory challenges and a significant workforce reduction. The therapy's efficacy comes amid scrutiny following patient deaths linked to liver injury, highlighting the risks associated with AAV therapies.

Regenxbio's RGX-202 gene therapy for Duchenne muscular dystrophy (DMD) demonstrated significant improvements in patient outcomes, exceeding expected disease progression at 12 and 18 months post-treatment in a pivotal trial. The company plans to submit a Biologics License Application (BLA) in mid-2026 via an accelerated approval pathway.

The HHS Secretary has accepted the recommendation to add Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel (RUSP), following public comments and evidence-based reports. This decision could enhance early detection and intervention for DMD, impacting patient outcomes significantly.