| Recruiting | A Study of Sepiapterin in Participants With Phenylketonuria (PKU) Phenylketonuria | Phase 3 | 2024-03-04 |
| Active Not Recruiting | A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia Friedreich Ataxia | Phase 3 | 2022-12-08 |
| Completed | A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Friedreich Ataxia | Phase 2 | 2022-10-12 |
| Completed | A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease Inherited Mitochondrial Disease | Phase 3 | 2022-06-22 |
| Completed | A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) Huntington Disease | Phase 2 | 2022-06-03 |
| Terminated | A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS) Leiomyosarcoma | Phase 2 / Phase 3 | 2022-05-23 |
| Terminated | Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS Amyotrophic Lateral Sclerosis | Phase 2 | 2022-05-15 |
| Active Not Recruiting | A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria Phenylketonuria | Phase 3 | 2022-02-14 |
| Completed | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Nonsene Mutation Duchenne Muscular Dystrophy | Phase 2 | 2021-12-29 |
| Completed | A Study of PTC923 in Participants With Phenylketonuria Phenylketonuria | Phase 3 | 2021-09-30 |
| Active Not Recruiting | A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exup AADC Deficiency | Phase 2 | 2021-05-12 |
| Completed | A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ata Friedreich Ataxia | Phase 2 / Phase 3 | 2020-12-17 |
| Terminated | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease | Phase 2 / Phase 3 | 2020-09-28 |
| Terminated | A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus Pneumonia, COVID-19, Coronavirus | Phase 2 / Phase 3 | 2020-07-09 |
| Terminated | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) Limb-Girdle Muscular Dystrophy | Phase 3 | 2019-10-31 |
| Completed | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Duchenne Muscular Dystrophy | Phase 2 | 2019-04-11 |
| Completed | A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LM Leiomyosarcoma | Phase 1 | 2019-03-13 |
| Completed | A Study of CNSA-001 in Women With Diabetic Gastroparesis Gastroparesis | Phase 2 | 2019-02-27 |
| Completed | A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalani BH4 Deficiency, Hyperphenylalaninemia | Phase 1 / Phase 2 | 2019-01-03 |
| Withdrawn | A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia Aniridia | Phase 2 | 2018-12-31 |
| Completed | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Duchenne Muscular Dystrophy | Phase 2 | 2018-12-21 |
| Withdrawn | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy | Phase 3 | 2018-10-31 |
| Terminated | Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias Leukemia, Myeloid, Acute, AML | Phase 1 | 2018-10-29 |
| Completed | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic | Phase 3 | 2017-07-06 |
| Completed | Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy | Phase 2 | 2016-06-09 |
| Completed | Study of Ataluren in Participants With Nonsense Mutation Aniridia Aniridia | Phase 2 | 2016-01-31 |
| Completed | Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS) Amyotrophic Lateral Sclerosis | Phase 2 | 2016-01-14 |
| Completed | PTC596 in Patients With Advanced Solid Tumors Cancer | Phase 1 | 2016-01-01 |
| Terminated | Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis Cystic Fibrosis | Phase 3 | 2015-08-31 |
| Completed | Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers Healthy Volunteers | Phase 1 | 2015-06-01 |
| Completed | Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Muscular Dystrophy, Duchenne | — | 2015-04-30 |
| Completed | Effects of Rifampin on the Pharmacokinetics of Ataluren Healthy | Phase 1 | 2015-02-01 |
| Completed | A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics Renal Impairment | Phase 1 | 2014-12-01 |
| Completed | A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics Hepatic Impairment | Phase 1 | 2014-12-01 |
| Completed | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrop Duchenne Muscular Dystrophy | Phase 1 | 2014-12-01 |
| Completed | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort Duchenne Muscular Dystrophy | Phase 1 | 2014-12-01 |
| Completed | Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Defla Healthy Volunteers | Phase 1 | 2014-11-01 |
| Completed | Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) Cystic Fibrosis | Phase 3 | 2014-08-01 |
| Terminated | Study of Ataluren (PTC124) in Cystic Fibrosis Cystic Fibrosis | Phase 3 | 2014-05-23 |
| Terminated | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic | Phase 3 | 2014-03-20 |
| Completed | Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome Leigh Syndrome | Phase 2 | 2014-01-31 |
| Completed | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic | Phase 3 | 2013-03-26 |
| Completed | Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia Friedreich's Ataxia | Phase 2 | 2012-12-31 |
| Completed | Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome Leigh Syndrome | Phase 2 | 2012-10-31 |
| Completed | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystroph Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy | Phase 3 | 2012-05-20 |
| Completed | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) Duchenne Muscular Dystrophy | Phase 3 | 2010-11-30 |
| Completed | Extension Study of Ataluren (PTC124) in Cystic Fibrosis Cystic Fibrosis | Phase 3 | 2010-08-12 |
| Terminated | Ataluren for Nonsense Mutation Methylmalonic Acidemia Amino Acid Metabolism, Inborn Errors | Phase 2 | 2010-07-19 |
| Terminated | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscu Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | 2010-01-13 |
| Terminated | Study of Ataluren (PTC124) in Hemophilia A and B Hemophilia A, Hemophilia B | Phase 2 | 2009-10-14 |
| Completed | Study of Ataluren (PTC124™) in Cystic Fibrosis Cystic Fibrosis | Phase 3 | 2009-09-08 |
| Terminated | PTC299 for Treatment of Neurofibromatosis Type 2 Neurofibromatosis 2 | Phase 2 | 2009-07-31 |
| Terminated | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | 2009-01-31 |
| Terminated | Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy | Phase 2 | 2008-08-13 |
| Terminated | PTC299 for Treatment of Advanced Cancer Advanced Cancer | Phase 1 | 2008-07-03 |
| Completed | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | 2008-02-29 |
| Completed | PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer Metastatic Breast Cancer | Phase 1 | 2007-11-01 |
| Completed | A Study of Ataluren in Pediatric Participants With Cystic Fibrosis Cystic Fibrosis | Phase 2 | 2007-03-23 |
| Completed | PTC124 for the Treatment of Cystic Fibrosis Cystic Fibrosis | Phase 2 | 2006-12-01 |
| Completed | Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy | Phase 2 | 2005-12-01 |
| Completed | Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis Cystic Fibrosis | Phase 2 | 2005-11-30 |
| Completed | PTC124 for Cystic Fibrosis Cystic Fibrosis | Phase 2 | 2005-09-01 |
| No Longer Available | EPI-743 for Mitochondrial Respiratory Chain Diseases Mitochondrial Diseases | — | — |
| Approved For Marketing | Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy | — | — |