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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: 76
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NCT IDTitle
NCT07614126Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene)
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT07681713Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT04014530Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
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)
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT04439071A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
NCT02139306Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT00351078PTC124 for the Treatment of Cystic Fibrosis
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT00234663PTC124 for Cystic Fibrosis
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT01910168A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT07779616Study of PTC844 in Participants With Active Rheumatoid Arthritis
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)