Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

Clinical Trial ID NCT01225211

Rank	Title	Journal	Year	PubWeight™‹?›
1	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.	Lancet Respir Med	2014	2.25
2	Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression.	Sci Transl Med	2014	1.64
3	State of progress in treating cystic fibrosis respiratory disease.	BMC Med	2012	0.96
4	Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.	Mol Pharmacol	2014	0.94
5	Fixing cystic fibrosis by correcting CFTR domain assembly.	J Cell Biol	2012	0.90
6	On the mechanism of gating defects caused by the R117H mutation in cystic fibrosis transmembrane conductance regulator.	J Physiol	2016	0.79
7	Nanotechnology approaches for inhalation treatment of fibrosis.	J Drug Target	2013	0.78
8	A genomic signature approach to rescue ΔF508-cystic fibrosis transmembrane conductance regulator biosynthesis and function.	Am J Respir Cell Mol Biol	2014	0.77
9	Interaction non grata between CFTR's correctors and potentiators.	Ann Transl Med	2015	0.76
10	Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.	Ann Am Thorac Soc	2016	0.75
11	CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis.	Front Pharmacol	2016	0.75
12	PharmGKB summary: very important pharmacogene information for CFTR.	Pharmacogenet Genomics	2015	0.75