About This Trial
AAV-based gene therapy delivering micro-dystrophin for ambulatory boys with DMD aged 4-7.
Primary Endpoints
- North Star Ambulatory Assessment change
- Micro-dystrophin expression
Latest Update
February 2026
52-week data shows significant improvement in muscle function. BLA submission expected Q3 2026.
Lead Investigators
DJ
Dr. Jerry Mendell Director, Center for Gene Therapy, Nationwide Children's