Overview of Ataluren (Translarna/PTC124) clinical trials

Trial publications Phase II open-label trial (NCT00264888) and extension (NCT00759876) Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) • Finkel RS, Flanigan KM, Wong B, et al. Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy. […]