Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease

Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study

(2019) John J McNamara et al.   Lancet Respiratory Medicine

Effectivenesss of ivacaftor in severe cystic fibrosis patients and non-G551D gating mutations

(2019) Donatello Salvatore et al.   PEDIATRIC PULMONOLOGY

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

(2019) Harry G M Heijerman et al.   LANCET

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience

(2018) Layla Diab-Cáceres et al.   ARCHIVOS DE BRONCONEUMOLOGIA

Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR

(2018) Jennifer L. Taylor-Cousar et al.   Journal of Cystic Fibrosis

Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience

(2018) Layla Diab-Cáceres et al.   ARCHIVOS DE BRONCONEUMOLOGIA

Ivacaftor treatment of cystic fibrosis in children aged 12 to

(2018) Margaret Rosenfeld et al.   Lancet Respiratory Medicine

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Dominic Keating et al.   NEW ENGLAND JOURNAL OF MEDICINE

Real-life acute lung function changes after lumacaftor/ivacaftor first administration in pediatric patients with cystic fibrosis

(2017) Aurélie Labaste et al.   Journal of Cystic Fibrosis

Immediate effects of lumacaftor/ivacaftor administration on lung function in patients with severe cystic fibrosis lung disease

(2017) Natalia Popowicz et al.   Journal of Cystic Fibrosis

Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease

(2017) Dominique Hubert et al.   Journal of Cystic Fibrosis

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

(2017) Steven M. Rowe et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

(2017) Jennifer L. Taylor-Cousar et al.   NEW ENGLAND JOURNAL OF MEDICINE

Growth in Prepubertal Children With Cystic Fibrosis Treated With Ivacaftor

(2017) Michael S. Stalvey et al.   PEDIATRICS

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

(2017) Felix Ratjen et al.   Lancet Respiratory Medicine

Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States

(2016) Jennifer Taylor-Cousar et al.   Journal of Cystic Fibrosis

Causes of death in French cystic fibrosis patients: The need for improvement in transplantation referral strategies!

(2016) Clémence Martin et al.   Journal of Cystic Fibrosis

Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis

(2016) J Stuart Elborn et al.   Lancet Respiratory Medicine

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effects of Ivacaftor in Patients With Cystic Fibrosis Who Carry the G551D Mutation and Have Severe Lung Disease

(2014) Peter J. Barry et al.   CHEST

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

(2014) Kris De Boeck et al.   Journal of Cystic Fibrosis

Effects of ivacaftor on severely ill patients with cystic fibrosis carrying a G551D mutation

(2013) Helge Hebestreit et al.   Journal of Cystic Fibrosis

The use of ivacaftor in an adult with severe lung disease due to cystic fibrosis (ΔF508/G551D)

(2013) Hari M. Polenakovik et al.   Journal of Cystic Fibrosis

Ivacaftor in a G551D Homozygote with Cystic Fibrosis

(2013) Michael J. Harrison et al.   NEW ENGLAND JOURNAL OF MEDICINE

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE