… of treatment with lumacaftor–ivacaftor was associated with … and adults with cystic fibrosis
homozygous for Phe508del who … Cystic fibrosis (CF) is a genetic disease caused by mutations …

… Lumacaftor-ivacaftor is a combination of two small molecule therapies targeting the basic
defect in cystic fibrosis (… Lumacaftor, the corrector, works by increasing the trafficking of CFTR …

… Children received lumacaftor 100 mg and ivacaftor 125 mg (bodyweight <14 kg) or …
manifestations of cystic fibrosis disease and the established lumacaftor and ivacaftor safety profile. …

… Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA)
in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and …

… evaluated the efficacy and safety of this lumacaftor–ivacaftor combined therapy in a real-life …
HRCT changes in cystic fibrosis patients treated with lumacaftor–ivacaftor. The secondary …

… is affected by cystic fibrosis is the lung, … lumacaftor and ivacaftor delivered by lipid nanoparticles
directly into the lungs was highly effective in treating lung manifestations of cystic fibrosis…

… lumacaftor (LUM) and potentiator ivacaftor (IVA) has been labeled in France since 2015 for
F508del homozygote cystic fibrosis (… of lumacaftor/ivacaftor in children with cystic fibrosis in …

… The results of this study support the ongoing clinical benefit of lumacaftor–ivacaftor for up
to 120 weeks to treat the underlying cause of cystic fibrosis in children aged 2 years or older …

… of patients: patients who received ivacaftor or lumacaftor/ivacaftor (which we refer to as
the … We hypothesized that the theratype of CF patients receiving ivacaftor or lumacaftor/ivacaftor …

Ivacaftor was approved by the US Food and Drug Administration in 2012 for patients with cystic
fibrosis … that targeted mutant CFTR (cystic fibrosis transmembrane regulator) protein, the …