Biological Characterization of F508delCFTR Protein Processing by the CFTR Corrector ABBV-2222/GLPG2222

Identifying the molecular target sites for CFTR potentiators GLPG1837 and VX-770

(2019) Han-I Yeh et al.   JOURNAL OF GENERAL PHYSIOLOGY

Ion Channel Modulators in Cystic Fibrosis

(2018) Martina Gentzsch et al.   CHEST

Therapeutic approaches to CFTR dysfunction: From discovery to drug development

(2018) Hongyu Li et al.   Journal of Cystic Fibrosis

CFTR modulator theratyping: Current status, gaps and future directions

(2018) John Paul Clancy et al.   Journal of Cystic Fibrosis

Structural mechanisms of CFTR function and dysfunction

(2018) Tzyh-Chang Hwang et al.   JOURNAL OF GENERAL PHYSIOLOGY

Discovery of 4-[(2R,4R)-4-({[1-(2,2-Difluoro-1,3-benzodioxol-5-yl)cyclopropyl]carbonyl}amino)-7-(difluoromethoxy)-3,4-dihydro-2H-chromen-2-yl]benzoic Acid (ABBV/GLPG-2222), a Potent Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Corrector for the Treatment of Cystic Fibrosis

(2018) Xueqing Wang et al.   JOURNAL OF MEDICINAL CHEMISTRY

Discovery of N-(3-Carbamoyl-5,5,7,7-tetramethyl-5,7-dihydro-4H-thieno[2,3-c]pyran-2-yl)-lH-pyrazole-5-carboxamide (GLPG1837), a Novel Potentiator Which Can Open Class III Mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Channels to a High Extent

(2018) Steven E. Van der Plas et al.   JOURNAL OF MEDICINAL CHEMISTRY

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

Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine

(2018) Nikhil T. Awatade et al.   Frontiers in Pharmacology

Effect of bronchodilators in healthy individuals receiving lumacaftor/ivacaftor combination therapy

(2017) Gautham Marigowda et al.   Journal of Cystic Fibrosis

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

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

A common mechanism for CFTR potentiators

(2017) Han-I Yeh et al.   JOURNAL OF GENERAL PHYSIOLOGY

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

Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor)

(2016) Elizabeth Matthes et al.   BRITISH JOURNAL OF PHARMACOLOGY

Fatty Acid Cysteamine Conjugates as Novel and Potent Autophagy Activators That Enhance the Correction of Misfolded F508del-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)

(2016) Chi B. Vu et al.   JOURNAL OF MEDICINAL CHEMISTRY

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

Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels

(2014) Z. Kopeikin et al.   Journal of Cystic Fibrosis

Cystic fibrosis genetics: from molecular understanding to clinical application

(2014) Garry R. Cutting   NATURE REVIEWS GENETICS

Some gating potentiators, including VX-770, diminish  F508-CFTR functional expression

(2014) G. Veit et al.   Science Translational Medicine

Potentiator ivacaftor abrogates pharmacological correction of  F508 CFTR in cystic fibrosis

(2014) D. M. Cholon et al.   Science Translational Medicine

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

(2013) Hong Yu Ren et al.   MOLECULAR BIOLOGY OF THE CELL

Mechanism-based corrector combination restores ΔF508-CFTR folding and function

(2013) Tsukasa Okiyoneda et al.   Nature Chemical Biology

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

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

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

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

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

(2011) Gergely L. Lukacs et al.   TRENDS IN MOLECULAR MEDICINE

Peripheral Protein Quality Control Removes Unfolded CFTR from the Plasma Membrane

(2010) T. Okiyoneda et al.   SCIENCE

Gating of the CFTR Cl−channel by ATP-driven nucleotide-binding domain dimerisation

(2009) Tzyh-Chang Hwang et al.   JOURNAL OF PHYSIOLOGY-LONDON

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