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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: 75
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NCT IDTitle
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT01370447EPI-743 for Mitochondrial Respiratory Chain Diseases
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT06254482An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT02409004Effects of Rifampin on the Pharmacokinetics of Ataluren
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT02286622A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT04439071A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT04336826A 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)
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT07159139Vatiquinone Expanded Access Protocol
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT04014530Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
NCT05358717A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
NCT02251600A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy