Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
Michael W. Konstan, Donald R. VanDevanter, Steven M. Rowe, M. Wilschanski, Eitan Kerem, Isabelle Sermet‐Gaudelus, Emily DiMango, Paola Melotti, Joseph McIntosh, K. De Boeck
Topics & Concepts
MedicineCystic fibrosisPlaceboInternal medicineRandomized controlled trialNonsense mutationPediatricsMutationPathologyAlternative medicineMissense mutationGeneticsBiologyGeneCystic Fibrosis Research AdvancesRespiratory viral infections researchBiological Research and Disease Studies