Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2018-02-09
Target enrollment:
Participant gender:
Summary
This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics
(PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with
Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.