Real world outcomes of CFTR modulator therapy in Australian adults and children

Rapid Improvement After Starting Elexacaftor-tezacaftor-ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease

(2021) Pierre-Régis Burgel et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Longitudinal effects of ivacaftor and medicine possession ratio in people with the Gly551Asp mutation: a 5-year study

(2021) Ruth Marian Mitchell et al.   THORAX

Review of CFTR modulators 2020

(2021) Danielle M. Goetz et al.   PEDIATRIC PULMONOLOGY

Long-Term Outcomes in Real Life of Lumacaftor–Ivacaftor Treatment in Adolescents With Cystic Fibrosis

(2021) Stéphanie Bui et al.   Frontiers in Pediatrics

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

(2020) Michal Shteinberg et al.   European Respiratory Review

Tezacaftor/ivacaftor in people with cystic fibrosis who stopped lumacaftor/ivacaftor due to respiratory adverse events

(2020) Carsten Schwarz et al.   Journal of Cystic Fibrosis

Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function

(2020) Pierre-Régis Burgel et al.   Journal of Cystic Fibrosis

Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for ≥24 Weeks in People With CF and ≥1 F508del Allele: Interim Results of an Open-Label Phase Three Clinical Trial

(2020) Matthias Griese et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

(2020) Renée V. E. Dagenais et al.   Journal of Clinical Medicine

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

P264 Real-world Orkambi cohort CorK study (ROCK) - a prospective twelve months' analysis addressing the effectiveness of CFTR modulation in patients with cystic fibrosis homozygous for F508del CFTR

(2019) P. Arooj et al.   Journal of Cystic Fibrosis

Longitudinal changes in lung function following initiation of lumacaftor/ivacaftor combination

(2019) Ioanna Loukou 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

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

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

Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp- CFTR mutation after 1 and 2 years of treatment with ivacaftor in a real-world setting

(2018) Dominique Hubert et al.   Journal of Cystic Fibrosis

Corticosteroid use and increased CXCR2 levels on leukocytes are associated with lumacaftor/ivacaftor discontinuation in cystic fibrosis patients homozygous for the F508del CFTR mutation

(2018) Kerstin Pohl et al.   PLoS One

Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation

(2017) Philip M. Farrell et al.   JOURNAL OF PEDIATRICS

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

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

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

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

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

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

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

(2013) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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