The Priority Review

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Duchenne Muscular Dystrophy

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  1. Clinical trials

    PTC Therapeutics posts results of Phase 3 ataluren safety study in Duchenne muscular dystrophy

    Results for the open-label safety study of ataluren (PTC124) in 265 participants with nonsense mutation dystrophinopathy were first posted on ClinicalTrials.gov on October 8, 2026. The study, which began on November 22, 2010 and reached primary completion on February 10, 2026, was terminated before completion.