Posted inNeurology news Pediatrics
CIFFREO Trial Results: Fordadistrogene Movaparvovec Fails Primary Efficacy Endpoint in Duchenne Muscular Dystrophy
The Phase 3 CIFFREO trial for the gene therapy fordadistrogene movaparvovec failed to meet its primary efficacy endpoint in boys with Duchenne muscular dystrophy, showing no significant functional improvement over placebo and leading to the discontinuation of its clinical development.