Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Key Points

• To evaluate the safety, tolerability, and biological efficacy of systemic intravenous infusion of rAAVrh74.MHCK7.micro-dystrophin in pediatric patients with Duchenne muscular dystrophy.
• Interventional clinical trial (ClinicalTrials.gov identifier: NCT03375164) assessing systemic intravenous delivery of rAAVrh74.MHCK7.micro-dystrophin gene therapy.
• Conducted in pediatric participants with confirmed Duchenne muscular dystrophy to assess micro-dystrophin expression and physical function.
• Primary assessment endpoints focus on vector safety, tolerability, and robust micro-dystrophin transgene expression in targeted muscle tissue.
• Long-term monitoring assesses changes in motor abilities and functional motor outcomes following gene transfer.