Clinical Trial TitlePhase 3 extension study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy
Clinical Trial Protocol Description:
This study is an extension of the Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy. The purpose of this study is to better understand whether Ataluren can be safely given for a long period of time.
The study will also provide additional information as to whether the actions of Ataluren can result in improved walking, activity, muscle function, muscle fragility, upper limb function, pulmonary function and health-related quality of life in boys with Duchenne muscular dystrophy.