Asitri Dashboard Search Clinical Trials
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: 76
Selected: 0
| NCT ID | Title | ||
|---|---|---|---|
| NCT04577352 | A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | ||
| NCT01826487 | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | ||
| NCT05166161 | A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria | ||
| NCT02758626 | Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome | ||
| NCT03648827 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | ||
| NCT02485431 | Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers | ||
| NCT01247207 | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) | ||
| NCT02460679 | Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS) | ||
| NCT00911248 | PTC299 for Treatment of Neurofibromatosis Type 2 | ||
| NCT00237380 | Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis | ||
| NCT00803205 | Study of Ataluren (PTC124™) in Cystic Fibrosis | ||
| NCT02286609 | A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics | ||
| NCT02404480 | PTC596 in Patients With Advanced Solid Tumors | ||
| NCT03761069 | Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias | ||
| NCT04378075 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | ||
| NCT02090959 | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy | ||
| NCT01721733 | Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome | ||
| NCT01910168 | A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis | ||
| NCT07614126 | Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB1 Gene) | ||
| NCT04336826 | A 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) | ||
| NCT01141075 | Ataluren for Nonsense Mutation Methylmalonic Acidemia | ||
| NCT02926066 | A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion | ||
| NCT00947193 | Study of Ataluren (PTC124) in Hemophilia A and B | ||
| NCT02107859 | Study of Ataluren (PTC124) in Cystic Fibrosis | ||
| NCT01728064 | Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia | ||
| NCT02251600 | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | ||
| NCT03712124 | A Study of CNSA-001 in Women With Diabetic Gastroparesis | ||
| NCT03605550 | A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade Glioma | ||
| NCT00458341 | A Study of Ataluren in Pediatric Participants With Cystic Fibrosis | ||
| NCT02456103 | Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis | ||
| NCT01009294 | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) | ||
| NCT00592553 | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | ||
| NCT00686842 | PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma | ||
| NCT03796637 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren | ||
| NCT06302348 | A Study of Sepiapterin in Participants With Phenylketonuria (PKU) | ||
| NCT03783923 | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | ||
| NCT00351078 | PTC124 for the Treatment of Cystic Fibrosis | ||
| NCT01140451 | Extension Study of Ataluren (PTC124) in Cystic Fibrosis | ||
| NCT05349721 | Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS | ||
| NCT03761095 | A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS) | ||
| NCT02409004 | Effects of Rifampin on the Pharmacokinetics of Ataluren | ||
| NCT05099640 | A Study of PTC923 in Participants With Phenylketonuria | ||
| NCT02647359 | Study of Ataluren in Participants With Nonsense Mutation Aniridia | ||
| NCT00847379 | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | ||
| NCT01370447 | EPI-743 for Mitochondrial Respiratory Chain Diseases | ||
| NCT02352896 | Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome | ||
| NCT01158300 | PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central Nervous System Tumors | ||
| NCT03519711 | A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia | ||
| NCT02369731 | Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | ||
| NCT07681713 | Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) |
