Long-term safety of lumacaftor–ivacaftor in children aged 2–5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label …

JE Hoppe, M Chilvers, F Ratjen… - The Lancet …, 2021 - thelancet.com
Background A previous phase 3 study showed that lumacaftor–ivacaftor was generally safe
and well tolerated over 24 weeks of treatment in children aged 2–5 years with cystic fibrosis …

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 …

MA Chilvers, JC Davies, C Milla, S Tian… - The Lancet …, 2021 - thelancet.com
Background The safety and efficacy of 24 weeks of lumacaftor–ivacaftor combination
therapy in children aged 6–11 years with cystic fibrosis homozygous for the F508del-CFTR …

Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for …

JJ McNamara, SA McColley, G Marigowda… - The Lancet …, 2019 - thelancet.com
Background The efficacy, safety, and tolerability of lumacaftor and ivacaftor are established
in patients aged 6 years and older with cystic fibrosis, homozygous for the F508del-CFTR …

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled …

F Ratjen, C Hug, G Marigowda, S Tian… - The lancet Respiratory …, 2017 - thelancet.com
Background Lumacaftor and ivacaftor combination treatment showed efficacy in patients
aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis …

A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR

JH Rayment, F Asfour, M Rosenfeld… - American journal of …, 2022 - atsjournals.org
Rationale: Previous phase 3 trials showed that treatment with lumacaftor/ivacaftor was safe
and efficacious in people aged⩾ 2 years with cystic fibrosis (CF) homozygous for the …

Real-life safety and effectiveness of lumacaftor–ivacaftor in patients with cystic fibrosis

PR Burgel, A Munck, I Durieu, R Chiron… - American journal of …, 2020 - atsjournals.org
Rationale: Lumacaftor–ivacaftor is a CFTR (cystic fibrosis transmembrane conductance
regulator) modulator combination recently approved for patients with cystic fibrosis (CF) …

Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del …

MW Konstan, EF McKone, RB Moss… - The lancet Respiratory …, 2017 - thelancet.com
Background The 24-week safety and efficacy of lumacaftor/ivacaftor combination therapy
was shown in two randomised controlled trials (RCTs)—TRAFFIC and TRANSPORT—in …

Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single …

JC Davies, S Cunningham, WT Harris… - The Lancet …, 2016 - thelancet.com
Background Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in
patients aged 6 years or older with a CFTR gating mutation. We aimed to assess the safety …

Lumacaftor/Ivacaftor in Patients Aged 6–11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR

CE Milla, F Ratjen, G Marigowda, F Liu… - American journal of …, 2017 - atsjournals.org
Rationale: Combination lumacaftor/ivacaftor has been shown to improve lung function and
other endpoints in patients aged 12 years and older with cystic fibrosis and homozygous for …

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis

M Talamo Guevara, SA McColley - Expert opinion on drug safety, 2017 - Taylor & Francis
Introduction: Lumacaftor-ivacaftor is indicated for treatment of cystic fibrosis (CF) in patients
homozygous for the Phe-508del cystic fibrosis transmembrane conductance regulator …