Fordadistrogene Movaparvovec Fails to Improve Functional Outcomes in Duchenne Muscular Dystrophy: Insights from the Phase 3 CIFFREO Trial

Fordadistrogene Movaparvovec Fails to Improve Functional Outcomes in Duchenne Muscular Dystrophy: Insights from the Phase 3 CIFFREO Trial

The Phase 3 CIFFREO trial found that the gene therapy fordadistrogene movaparvovec did not significantly improve motor function in boys with Duchenne muscular dystrophy compared to placebo, leading to the discontinuation of its clinical development due to a negative benefit-risk profile.