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Long-term safety and efficacy of lumacaftor–ivacaftor therapy in children aged 6–11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study

Mark Chilvers, Jane C. Davies, Carlos Milla, Simon Tian, Zifei Han, Alexandra G. Cornell, Caroline A. Owen, Félix Ratjen

2021The Lancet Respiratory Medicine42 citationsDOI

Topics & Concepts

IvacaftorTolerabilityCystic fibrosisMedicinePlaceboInternal medicineCystic fibrosis transmembrane conductance regulatorClinical endpointAdverse effectClinical trialPathologyAlternative medicineCystic Fibrosis Research AdvancesNeonatal Respiratory Health ResearchPediatric health and respiratory diseases
Long-term safety and efficacy of lumacaftor–ivacaftor therapy in children aged 6–11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study | Litcius