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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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NCT IDTitle
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02819557Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT01370447EPI-743 for Mitochondrial Respiratory Chain Diseases
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
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
NCT07614126Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene)
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT07779616Study of PTC844 in Participants With Active Rheumatoid Arthritis
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT00234663PTC124 for Cystic Fibrosis
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
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
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants