Effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease with the Phe508del/minimal function genotype

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

Reply to: Optimism with Caution: Elexacaftor-Tezacaftor-Ivacaftor in Patients with Advanced Pulmonary Disease

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

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

(2020) Michal Shteinberg et al.   European Respiratory Review

Ivacaftor improves lung disease in patients with advanced CF carrying CFTR mutations that confer residual function

(2020) Donatello Salvatore et al.   RESPIRATORY MEDICINE

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

Projecting the impact of delayed access to elexacaftor/tezacaftor/ivacaftor for people with Cystic Fibrosis

(2020) Sanja Stanojevic et al.   Journal of Cystic Fibrosis

Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination

(2020) Guido Veit et al.   JCI Insight

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

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

Multicenter validation study for the certification of a CFTR gene scanning method using next generation sequencing technology

(2018) Anne Bergougnoux et al.   CLINICAL CHEMISTRY AND LABORATORY 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

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

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

Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis

(2016) Caitlyn T. Solem et al.   Health and Quality of Life Outcomes

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

(2016) Gudio Veit et al.   MOLECULAR BIOLOGY OF THE CELL

Progress in therapies for cystic fibrosis

(2016) Kris De Boeck 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

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

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

Validation of a predictive survival model in Italian patients with cystic fibrosis

(2011) Roberto Buzzetti et al.   Journal of Cystic Fibrosis

Extensive Molecular Analysis of Patients Bearing CFTR-Related Disorders

(2011) Felice Amato et al.   JOURNAL OF MOLECULAR DIAGNOSTICS

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

Failure to Recover to Baseline Pulmonary Function after Cystic Fibrosis Pulmonary Exacerbation

(2010) Don B. Sanders et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Use of the Peak Heart Rate Reached During Six-Minute Walk Test to Predict Individualized Training Intensity in Patients With Cystic Fibrosis: Validity and Reliability

(2010) Mathieu Gruet et al.   ARCHIVES OF PHYSICAL MEDICINE AND REHABILITATION