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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
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT07681713Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT02409004Effects of Rifampin on the Pharmacokinetics of Ataluren
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT00508586PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT01910168A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT02295748An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
NCT02647359Study of Ataluren in Participants With Nonsense Mutation Aniridia
NCT05166161A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
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
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT02090959An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
NCT01141075Ataluren for Nonsense Mutation Methylmalonic Acidemia
NCT00759876Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT07779616Study of PTC844 in Participants With Active Rheumatoid Arthritis
NCT02286635Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single Dose Pharmacokinetics of Deflazacort
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT02460679Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT07614126Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene)
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT00351078PTC124 for the Treatment of Cystic Fibrosis
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)