Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?

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

Development of HPLC and LC–MS/MS methods for the analysis of ivacaftor, its major metabolites and lumacaftor in plasma and sputum of cystic fibrosis patients treated with ORKAMBI or KALYDECO

(2016) Elena K. Schneider et al.   JOURNAL OF CHROMATOGRAPHY B-ANALYTICAL TECHNOLOGIES IN THE BIOMEDICAL AND LIFE SCIENCES

CFTR modulators and pregnancy: Our work has only just begun

(2016) Christopher H. Goss et al.   Journal of Cystic Fibrosis

A successful uncomplicated CF pregnancy while remaining on Ivacaftor

(2016) Rachel Kaminski et al.   Journal of Cystic Fibrosis

An “Unlikely” Pair: The Antimicrobial Synergy of Polymyxin B in Combination with the Cystic Fibrosis Transmembrane Conductance Regulator Drugs KALYDECO and ORKAMBI

(2016) Elena K. Schneider et al.   ACS Infectious Diseases

F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review

(2015) Hong Yang et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Drug-drug plasma protein binding interactions of ivacaftor

(2015) Elena K. Schneider et al.   JOURNAL OF MOLECULAR RECOGNITION

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

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

Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?

(2015) A. M. Jones et al.   THORAX

Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction

(2015) John J. Brewington et al.   Expert Review of Respiratory Medicine

Impact of the CFTR-Potentiator Ivacaftor on Airway Microbiota in Cystic Fibrosis Patients Carrying A G551D Mutation

(2015) Cédric Bernarde et al.   PLoS One

Longevity of Patients With Cystic Fibrosis in 2000 to 2010 and Beyond: Survival Analysis of the Cystic Fibrosis Foundation Patient Registry

(2014) Todd MacKenzie et al.   ANNALS OF INTERNAL MEDICINE

Functional and pharmacological induced structural changes of the cystic fibrosis transmembrane conductance regulator in the membrane solved using SAXS

(2014) Debora Baroni et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

VX-809 and Related Corrector Compounds Exhibit Secondary Activity Stabilizing Active F508del-CFTR after Its Partial Rescue to the Cell Surface

(2014) Paul D.W. Eckford et al.   CHEMISTRY & BIOLOGY

Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis

(2014) Penny Whiting et al.   HEALTH TECHNOLOGY ASSESSMENT

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

Antibacterial properties of the CFTR potentiator ivacaftor

(2014) Leah R. Reznikov et al.   Journal of Cystic Fibrosis

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

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

Ivacaftor: A Review of Its Use in Patients with Cystic Fibrosis

(2013) Emma D. Deeks   DRUGS

Repairing mutated proteins – development of small molecules targeting defects in the cystic fibrosis transmembrane conductance regulator

(2013) Daniel Merk et al.   Expert Opinion on Drug Discovery

Sweat chloride as a biomarker of CFTR activity: Proof of concept and ivacaftor clinical trial data

(2013) Frank J. Accurso et al.   Journal of Cystic Fibrosis

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

Understanding the Costs of Care for Cystic Fibrosis: An Analysis by Age and Health State

(2013) Kees van Gool et al.   VALUE IN HEALTH

A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect

(2013) Michael P Boyle et al.   Lancet Respiratory Medicine

Identification and Validation of Hits from High Throughput Screens for CFTR Modulators

(2012) Stan Pasyk et al.   CURRENT PHARMACEUTICAL DESIGN

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner

(2012) Paul D. W. Eckford et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Targeting the regulation of CFTR channels

(2011) Paul D.W. Eckford et al.   BIOCHEMICAL JOURNAL

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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

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

Thermal unfolding studies show the disease causing F508del mutation in CFTR thermodynamically destabilizes nucleotide-binding domain 1

(2010) Irina Protasevich et al.   PROTEIN SCIENCE

Integrated biophysical studies implicate partial unfolding of NBD1 of CFTR in the molecular pathogenesis of F508del cystic fibrosis

(2010) Chi Wang et al.   PROTEIN SCIENCE

Hepatobiliary disease in patients with cystic fibrosis

(2009) Katie Moyer et al.   CURRENT OPINION IN GASTROENTEROLOGY

Chloride channels as drug targets

(2009) Alan S. Verkman et al.   NATURE REVIEWS DRUG DISCOVERY

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

The Clinical Approach to Lung Disease in Patients with Cystic Fibrosis

(2009) Brian O'Sullivan et al.   SEMINARS IN RESPIRATORY AND CRITICAL CARE MEDICINE

The prevalence of cystic fibrosis in the European Union

(2008) Philip M. Farrell   Journal of Cystic Fibrosis

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

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

High treatment burden in adults with cystic fibrosis: Challenges to disease self-management

(2008) Gregory S. Sawicki et al.   Journal of Cystic Fibrosis