Dystrophinopathy

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CLINICAL_TRIALS trial_result · 2020-11-25
Clinical Trial Update: Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada [Phase 3]
Phase 3 trial update for ataluren in previously treated nmDBMD participants with nonsense mutation Duchenne/Becker muscular dystrophy; study…