Randomized Trialhigh evidence
Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study.
The Lancet. Neurology·March 1, 2026·PMID 41722591
Why it mattersThis is the first phase 3 report of an AAV9 mini-dystrophin gene therapy in Duchenne muscular dystrophy. The negative result and safety concerns inform expectations for this class of therapies and highlight the challenges of translating early-phase promise into functional benefit.
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