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Dystrophinopathy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Dystrophinopathy trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Dystrophinopathy trials may be worth asking aboutNorth America
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The goal of this clinical trial is to assess the effects of a dual-task, multimodal training program on slowing functional motor decline in children and adolesc…
This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) thr…
The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the…
The goal of this observational study is to investigate whether the type, location, and extent of pathogenic variants in the DMD gene are associated with cardiac…
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had…