An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB).
GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1)
Rate of Uptake of Ivacaftor Use after U.S. Food and Drug Administration Approval among Patients Enrolled in the U.S. Cystic Fibrosis Foundation Patient Registry
Disease Progression in Patients with Cystic Fibrosis Treated with Ivacaftor: Data From National US and UK Registries
Italian and North American dietary intake after ivacaftor treatment for Cystic Fibrosis Gating Mutations.
Human epididymis protein 4 (HE4) levels inversely correlate with lung function improvement (delta FEV1) in cystic fibrosis patients receiving ivacaftor treatment.
Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV1.
Measured fetal and neonatal exposure to Lumacaftor and Ivacaftor during pregnancy and while breastfeeding
Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR
