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)
- Status
- COMPLETED
- Phase
- PHASE2
Clinical Trials Related to Ataluren
Related studies linked through entity extraction aliases.
A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
- Status
- WITHDRAWN
- Phase
- PHASE2
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
- Status
- COMPLETED
- Phase
- PHASE2
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Status
- COMPLETED
- Phase
- PHASE2
PTC Study to Evaluate Ataluren in Combination With Ivacaftor
- Status
- COMPLETED
- Phase
- PHASE4
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
- Status
- COMPLETED
- Phase
- PHASE3
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
- Status
- COMPLETED
- Phase
- PHASE2
Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
- Status
- COMPLETED
- Phase
- PHASE2
Study of Ataluren in Participants With Nonsense Mutation Aniridia
- Status
- COMPLETED
- Phase
- PHASE2
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
- Status
- TERMINATED
- Phase
- PHASE3
Effects of Rifampin on the Pharmacokinetics of Ataluren
- Status
- COMPLETED
- Phase
- PHASE1
Study of Ataluren (PTC124) in Cystic Fibrosis
- Status
- TERMINATED
- Phase
- PHASE3
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
- Status
- TERMINATED
- Phase
- PHASE3
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- Status
- COMPLETED
- Phase
- PHASE3
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
- Status
- COMPLETED
- Phase
- PHASE3
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
- Status
- COMPLETED
- Phase
- PHASE3
Ataluren for Nonsense Mutation Methylmalonic Acidemia
- Status
- TERMINATED
- Phase
- PHASE2
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
- Status
- COMPLETED
- Phase
- PHASE3
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
- Status
- TERMINATED
- Phase
- PHASE2
Study of Ataluren (PTC124) in Hemophilia A and B
- Status
- TERMINATED
- Phase
- PHASE2