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CIFFREO Trial

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Neurology

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 dev

Mar 8, 2026
Neurology

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

Feb 24, 2026

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