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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
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT07159139Vatiquinone Expanded Access Protocol
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT01009294Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT02139306Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT00234663PTC124 for Cystic Fibrosis
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT01910168A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
NCT00351078PTC124 for the Treatment of Cystic Fibrosis
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
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)
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT05358717A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT04439071A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT00704821PTC299 for Treatment of Advanced Cancer
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)
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT04014530Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis