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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
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
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)
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT02409004Effects of Rifampin on the Pharmacokinetics of Ataluren
NCT07159139Vatiquinone Expanded Access Protocol
NCT00234663PTC124 for Cystic Fibrosis
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
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
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01370447EPI-743 for Mitochondrial Respiratory Chain Diseases
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome