Treatment of Cystic Fibrosis Patients Homozygous for F508del with Lumacaftor-Ivacaftor (Orkambi®) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells

Predictive factors for lumacaftor/ivacaftor clinical response

(2018) Alexandra Masson et al.   Journal of Cystic Fibrosis

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have differential effects on cystic fibrosis macrophage function

(2018) Shuzhong Zhang et al.   Scientific Reports

Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation

(2017) Xin Meng et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

CFTR-dependent chloride efflux in cystic fibrosis mononuclear cells is increased by ivacaftor therapy

(2017) Lorenzo Guerra et al.   PEDIATRIC PULMONOLOGY

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

Neutrophil Membrane Cholesterol Content is a Key Factor in Cystic Fibrosis Lung Disease

(2017) Michelle M. White et al.   EBioMedicine

Ivacaftor-Induced Proteomic Changes Suggest Monocyte Defects May Contribute to the Pathogenesis of Cystic Fibrosis

(2016) Katherine B. Hisert et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

(2016) Johanna F. Dekkers et al.   Science Translational Medicine

Antibiotic therapy affects functional behaviour in cystic fibrosis blood mononuclear cells

(2015) Lorenzo Guerra et al.   EUROPEAN RESPIRATORY JOURNAL

Mechanical thrombectomy in acute ischemic stroke: Consensus statement by ESO-Karolinska Stroke Update 2014/2015, supported by ESO, ESMINT, ESNR and EAN

(2015) Nils Wahlgren et al.   International Journal of Stroke

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

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

Cystic fibrosis

(2015) Felix Ratjen et al.   Nature Reviews Disease Primers

A neutrophil intrinsic impairment affecting Rab27a and degranulation in cystic fibrosis is corrected by CFTR potentiator therapy

(2014) K. Pohl et al.   BLOOD

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

Potentiator ivacaftor abrogates pharmacological correction of  F508 CFTR in cystic fibrosis

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

Optimal Complement-Mediated Phagocytosis ofPseudomonas aeruginosaby Monocytes Is Cystic Fibrosis Transmembrane Conductance Regulator–Dependent

(2013) Pauline B. Van de Weert–van Leeuwen et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

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

Absence of the cystic fibrosis transmembrane regulator (Cftr ) from myeloid-derived cells slows resolution of inflammation and infection

(2012) T. L. Bonfield et al.   JOURNAL OF LEUKOCYTE BIOLOGY

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

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