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A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation

Jane C. Davies, Isabelle Sermet‐Gaudelus, Lutz Naehrlich, R. Scott Harris, Daniel Campbell, Neil Ahluwalia, Christopher Short, Eric L. Haseltine, Paul Panorchan, Clare Saunders, Caroline A. Owen, Claire Wainwright

2020Journal of Cystic Fibrosis68 citationsDOI

Topics & Concepts

IvacaftorMedicineCystic fibrosisPlaceboInternal medicineDiscontinuationAdverse effectCystic fibrosis transmembrane conductance regulatorPathologyAlternative medicineCystic Fibrosis Research AdvancesDelphi Technique in ResearchPediatric health and respiratory diseases
A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation | Litcius