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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
NCT01557400Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
NCT01140451Extension Study of Ataluren (PTC124) in Cystic Fibrosis
NCT02409004Effects of Rifampin on the Pharmacokinetics of Ataluren
NCT03206645Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
NCT02139306Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
NCT03605550A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma
NCT00264888Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
NCT02107859Study of Ataluren (PTC124) in Cystic Fibrosis
NCT01728064Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT03519711A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
NCT00234663PTC124 for Cystic Fibrosis
NCT02352896Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT01158300PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors
NCT05218655A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
NCT05485987A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT05515536A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT01826487Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT00803205Study of Ataluren (PTC124™) in Cystic Fibrosis
NCT01009294Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT00686842PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma
NCT00911248PTC299 for Treatment of Neurofibromatosis Type 2
NCT04378075A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
NCT02251600A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT00592553Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02286622A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics
NCT00847379Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT02592941Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT02286609A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics
NCT02758626Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome
NCT02456103Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
NCT01395641A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
NCT03761069Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
NCT03783923A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NCT01247207Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
NCT05349721Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT02404480PTC596 in Patients With Advanced Solid Tumors
NCT05269355A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)
NCT05358717A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
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
NCT04577352A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
NCT00947193Study of Ataluren (PTC124) in Hemophilia A and B
NCT03648827A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT00458341A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
NCT02485431Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
NCT01370447EPI-743 for Mitochondrial Respiratory Chain Diseases
NCT03796637A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
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)
NCT06302348A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NCT01721733Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome