Failure of Pfizer against Duchenne muscular dystrophy


(AOF) – Pfizer announced the failure of Ciffreo, a global phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluating fordadistrogen movaparvovec, its investigational mini-dystrophin gene therapy, in patients ambulatory patients with Duchenne muscular dystrophy (DMD). The study did not meet its primary endpoint of improvement in motor function in boys aged 4 to 7 years compared to placebo.

Key secondary endpoints, including 10-meter run/walk speed and time to rise from ground speed, also showed no significant difference from placebo.

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