Found 6 matching trials
Ataluren for Nonsense Mutation Methylmalonic Acidemia PHASE2 Methylmalonic acidemia (MMA) is a rare genetic disorder caused by mutations...
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ZNA Queen Paola Child Hospital and Provincial Centre for Metabolic Disorders
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Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) PHASE3 This is a Phase 3, international, multicenter, randomized, double-blind, pl...
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University of Alabama at Birmingham
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Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada PHASE3 Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develo...
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Royal Children's Hospital
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Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy PHASE2 This is a Phase 2, multiple-dose, open-label study evaluating the safety, p...
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Extension Study of Ataluren (PTC124) in Cystic Fibrosis PHASE3 Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene...
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University of Alabama-Birmingham
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Effects of Rifampin on the Pharmacokinetics of Ataluren PHASE1 This will be a single centre, Phase I, open-label, one cohort, one dose lev...
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