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A phase 3, randomized, double - blind, placebo - controlled efficacy and safety study of ataluren in patients with nonsense mutation Duchenne Muscular Dystrophy and open - label extension.
DMD_AtalurenRCT41
What is the goal of the study?
This is a randomized controlled trial of ambulatory boys ages 5 years and older with Duchenne Muscular Dystrophy (DMD) due to a nonsense mutation in the dystrophin gene, with boys receiving either placebo or ataluren for 72 weeks, then receiving ataluren for 72 weeks. Boys have follow up visits during the study including physical therapy testing, respiratory and cardiac monitoring, and physical exams.
Who can participate in the study?
Everyone