Overview

• The American Academy of Neurology released an Evidence in Focus article evaluating delandistrogene moxeparvovec, the first FDA-approved gene therapy for Duchenne muscular dystrophy.
• Phase 3 clinical trials failed to meet primary motor function endpoints, raising questions about the therapy’s efficacy in altering disease progression.
• The therapy carries serious safety risks, including muscle inflammation, myocarditis, thrombocytopenia, liver injury, and rare cases of death, necessitating close monitoring.
• The one-time infusion costs $3.2 million, excluding additional hospital and follow-up expenses, posing accessibility challenges for patients and families.
• Experts emphasize the need for long-term studies to assess potential benefits, safety, and the therapy's impact on quality of life and survival.