Clinical Trials – Muscular Dystrophy in Children

Drug Treatment Study for Boys with Duchenne Muscular Dystrophy

A multi-site, randomized, placebo-controlled, double-blind, multiple ascending subcutaneous dose study to evaluate the safety, tolerability and pharmcokinetics of BMS-986089 in ambulatory boys with Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy Treatment Study

Open-label, multiple-dose, efficacy, safety, and tolerability study of Eteplirsen in subjects with Duchenne muscular dystrophy who participated in study 4658-us-201

Extension Study of Ataluren Treatment for Nonsense Mutation Dystrophinopathy

Phase 3 extension study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy

Nonsense Mutation Dystrophinopathy and Ataluren Treatment Study

Phase 3 efficacy and safety study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy