PTC Therapeutics

NCT00234663

PTC124 for Cystic Fibrosis

NCT00237380

Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

NCT00264888

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

NCT00351078

PTC124 for the Treatment of Cystic Fibrosis

NCT00458341

A Study of Ataluren in Pediatric Participants With Cystic Fibrosis

NCT00592553

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

NCT00759876

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

NCT00847379

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

NCT00911248

PTC299 for Treatment of Neurofibromatosis Type 2

NCT00947193

Study of Ataluren (PTC124) in Hemophilia A and B

NCT01009294

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

NCT01141075

Ataluren for Nonsense Mutation Methylmalonic Acidemia

NCT01721733

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

NCT01728064

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

NCT02352896

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

NCT02460679

Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT02647359

Study of Ataluren in Participants With Nonsense Mutation Aniridia

NCT02819557

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

NCT02926066

A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion

NCT02758626

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

NCT03648827

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

NCT04117880

A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

NCT03712124

A Study of CNSA-001 in Women With Diabetic Gastroparesis

NCT03796637

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

NCT04903288

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

NCT04336826

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)

NCT05358717

A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

NCT05349721

Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

NCT05485987

A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

NCT06254482

An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)