Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopa
Brief Summary: This is a Phase 2, multiple-dose, open-label study evaluating the safety, and pharmacokinetics (PK) of ataluren in patients aged ≥2 to <5 years old with Duchenne muscular dystrophy caused by a nonsense mutation in the dystrophin gene. The study includes a 4-week screening period, a 4-week study period, and a 48-week extension period for patients who complete the 4-week study period (52 weeks total treatment).