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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
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT07614126Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene)
NCT07159139Vatiquinone Expanded Access Protocol
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT07681713Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT02139306Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT04439071A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT00351078PTC124 for the Treatment of Cystic Fibrosis
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT00234663PTC124 for Cystic Fibrosis
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis