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

Improvement of Hepatic Steatosis in Cystic Fibrosis With Ivacaftor Therapy

(2015) Don Hayes et al.   JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

(2014) Steven M. Rowe et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Effects of Ivacaftor in Patients With Cystic Fibrosis Who Carry the G551D Mutation and Have Severe Lung Disease

(2014) Peter J. Barry et al.   CHEST

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

Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp- CFTR mutation: a phase 3, open-label extension study (PERSIST)

(2014) Edward F McKone et al.   Lancet Respiratory Medicine

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

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

Aquagenic Wrinkling of the Palms in a Patient with Cystic Fibrosis

(2013) Hartmut Grasemann et al.   NEW ENGLAND JOURNAL OF MEDICINE

Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study

(2013) Melena D Bellin et al.   PEDIATRIC DIABETES

Sweat chloride is not a useful marker of clinical response to Ivacaftor

(2013) Peter J Barry et al.   THORAX

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

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