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The U.S. National Library of Medicine provides a database of clinical trials worldwide. Use the fields below to search the database. Expand your results and select terms or phrases and right-click to search using Google, MalaCards, MedlinePlus, PubMed, or Wikipedia.

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Clinical Trials Search Results

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Count: 74
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NCT IDTitle
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT01009294Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT01370447EPI-743 for Mitochondrial Respiratory Chain Diseases
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT02251600A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT05358717A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00351078PTC124 for the Treatment of Cystic Fibrosis
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)