A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant
Gregory S. Sawicki, Mark Chilvers, John McNamara, Lutz Naehrlich, Clare Saunders, Isabelle Sermet‐Gaudelus, Claire Wainwright, Neil Ahluwalia, Daniel Campbell, R. Scott Harris, Hildegarde Paz-Díaz, Judy L. Shih, Jane C. Davies
Topics & Concepts
IvacaftorTolerabilityMedicineCystic fibrosisDiscontinuationAdverse effectInternal medicineClinical endpointClinical trialCystic fibrosis transmembrane conductance regulatorCystic Fibrosis Research AdvancesPediatric health and respiratory diseasesAsthma and respiratory diseases