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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
NCT04117880A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT03642145A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT04439071A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)
NCT07159139Vatiquinone Expanded Access Protocol
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
NCT00234663PTC124 for Cystic Fibrosis
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02369731Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT03761095A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02286622A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02926066A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
NCT03712124A Study of CNSA-001 in Women With Diabetic Gastroparesis
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
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
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT00237380Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT00704821PTC299 for Treatment of Advanced Cancer
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT04903288A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
NCT05099640A Study of PTC923 in Participants With Phenylketonuria
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT03179631Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
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
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT01910168A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis