Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study

Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations

(2020) Anne Munck et al.   Journal of Cystic Fibrosis

A phase 3, randomized, double-blind, parallel-group study to evaluate tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for F508del-CFTR and a gating mutation

(2020) Edward F. McKone et al.   Journal of Cystic Fibrosis

Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries

(2019) Nataliya Volkova et al.   Journal of Cystic Fibrosis

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

Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor

(2018) Leona Bessonova et al.   THORAX

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

Patients with cystic fibrosis having a residual function mutation: Data from the Italian registry

(2018) Donatello Salvatore et al.   PEDIATRIC PULMONOLOGY

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

Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study

(2017) Michael W Konstan et al.   Lancet Respiratory Medicine

Cystic fibrosis

(2016) J Stuart Elborn   LANCET

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

Progress in therapies for cystic fibrosis

(2016) Kris De Boeck et al.   Lancet Respiratory Medicine

Sustained Benefit from Ivacaftor Demonstrated by Combining Clinical Trial and Cystic Fibrosis Patient Registry Data

(2015) Gregory S. Sawicki et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Idiosyncratic reactions are the most common cause of abnormal liver function tests in patients with cystic fibrosis

(2015) T. Jong et al.   INTERNAL MEDICINE JOURNAL

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

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

Cystic fibrosis: An update for clinicians. Part 2: Hepatobiliary and pancreatic manifestations

(2014) Oren Ledder et al.   JOURNAL OF GASTROENTEROLOGY AND HEPATOLOGY

Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp- CFTR mutation: a phase 3, open-label extension study (PERSIST)

(2014) Edward F McKone et al.   Lancet Respiratory Medicine

Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equations

(2012) Philip H. Quanjer et al.   EUROPEAN RESPIRATORY JOURNAL

Effect of pulmonary exacerbations on long-term lung function decline in cystic fibrosis

(2011) Valerie Waters et al.   EUROPEAN RESPIRATORY JOURNAL

Hepatic safety of antibiotics used in primary care

(2011) R. J. Andrade et al.   JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis

(2011) K. de Boer et al.   THORAX

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Clinical phenotype of cystic fibrosis patients with the G551D mutation

(2009) D.M. Comer et al.   QJM-AN INTERNATIONAL JOURNAL OF MEDICINE

Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice

(2008) C. Castellani et al.   Journal of Cystic Fibrosis