From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis

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

(2020) Michal Shteinberg et al.   European Respiratory Review

GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1)

(2019) Jane C. Davies et al.   Journal of Cystic Fibrosis

CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials

(2019) Scott C. Bell et al.   Journal of Cystic Fibrosis

A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis

(2019) Seth Walker et al.   Journal of Cystic Fibrosis

WS06-2 Initial results evaluating the add-on effect of the novel CFTR corrector PTI-801 in cystic fibrosis subjects

(2019) M. Jain et al.   Journal of Cystic Fibrosis

A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis

(2019) Al-Rahim R. Habib et al.   Scientific Reports

The future of cystic fibrosis care: a global perspective

(2019) Scott C Bell et al.   Lancet Respiratory Medicine

GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN)

(2019) Silke van Koningsbruggen-Rietschel 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

Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR

(2018) Scott H. Donaldson et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

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

(2018) Leona Bessonova et al.   THORAX

Peripheral Protein Quality Control as a Novel Drug Target for CFTR Stabilizer

(2018) Ryosuke Fukuda et al.   Frontiers in Pharmacology

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

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Jane C. Davies et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pharmacokinetics and safety of cavosonstat (N91115) in healthy and cystic fibrosis adults homozygous for F 508 DEL - CFTR

(2017) Scott H. Donaldson 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

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

Use of a High-Throughput Phenotypic Screening Strategy to Identify Amplifiers, a Novel Pharmacological Class of Small Molecules That Exhibit Functional Synergy with Potentiators and Correctors

(2017) Kenneth A. Giuliano et al.   SLAS Discovery

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 who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial

(2015) Richard B Moss et al.   Lancet Respiratory 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

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

(2014) Eitan Kerem et al.   Lancet Respiratory Medicine

A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial

(2014) Michael P Boyle et al.   Lancet Respiratory Medicine

Preferred Reporting Items for Systematic Reviews and Meta-Analyses: The PRISMA Statement

(2013) David Moher   ANNALS OF INTERNAL MEDICINE

Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D- CFTR mutation and preserved spirometry: a randomised controlled trial

(2013) Jane Davies et al.   Lancet Respiratory Medicine

Mechanism of Inhibition for N6022, a First-in-Class Drug Targeting S-Nitrosoglutathione Reductase

(2012) Louis S. Green et al.   BIOCHEMISTRY

Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

(2012) Patrick A. Flume et al.   CHEST

Targeting F508del-CFTR to develop rational new therapies for cystic fibrosis

(2011) Zhi-wei Cai et al.   ACTA PHARMACOLOGICA SINICA

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation

(2011) J P Clancy et al.   THORAX

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE