Ataluren

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

Study of Ataluren in Participants With Nonsense Mutation Aniridia

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Ataluren for Nonsense Mutation Methylmalonic Acidemia

Study of Ataluren (PTC124) in Hemophilia A and B

Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

Study of Ataluren (PTC124™) in Cystic Fibrosis

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis