Rare DiseasePfizer
Pfizer Reports Phase 1b Results for Gene Therapy in Duchenne Muscular Dystrophy
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Biotechnology Media Insights
Editorial Team · May 18, 2025
Pfizer announces Phase 1b results for fordadistrogene movaparvovec gene therapy showing micro-dystrophin expression and functional improvements in Duchenne muscular dystrophy.
Pfizer today announced Phase 1b results for fordadistrogene movaparvovec, an investigational gene therapy for Duchenne muscular dystrophy (DMD). The data showed robust micro-dystrophin expression in muscle tissue and improvements in functional assessments including the North Star Ambulatory Assessment.
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