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VX 809

Known as: VX-809 
National Institutes of Health

Related topics

Related topics

1 relation

Broader (1)

lumacaftor

Papers overview

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2020
2020
Structural Consequences of the 1,2,3‐Triazole as an Amide Bioisostere in Analogues of the Cystic Fibrosis Drugs VX‐809 and VX‐770
Although the 1,2,3‐triazole is a commonly used amide bioisostere in medicinal chemistry, the structural implications of this… 
2019
2019
Pharmacological reversal of renal cysts from secretion to absorption suggests a potential therapeutic strategy for managing autosomal dominant polycystic kidney disease
Autosomal-dominant polycystic kidney disease (ADPKD) induces a secretory phenotype, resulting in multiple fluid-filled cysts. We… 
2019
2019
Activity of lumacaftor is not conserved in zebrafish Cftr bearing the major cystic fibrosis‐causing mutation
F508del‐cystic fibrosis transmembrane conductance regulator (CFTR) is the major mutant responsible for cystic fibrosis (CF… 
2018
2018
Structural mechanisms for defective CFTR gating caused by the Q1412X mutation, a severe Class VI pathogenic mutation in cystic fibrosis
Electrophysiological characterization of Q1412X‐CFTR, a C‐terminal truncation mutation of cystic fibrosis transmembrane… 
2017
2017
Direct Measurement of Trafficking of the Cystic Fibrosis Transmembrane Conductance Regulator to the Cell Surface and Binding to a Chemical Chaperone.
Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) result in the disease cystic fibrosis. Deletion of… 
2016
2016
Ion Channels and Transporters in Lung Function and Disease Restoration of R 117 H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770
Martina Gentzsch,* Hong Y. Ren, Scott A. Houck, Nancy L. Quinney, Deborah M. Cholon, Pattarawut Sopha, Imron G. Chaudhry, Jhuma… 
2016
2016
Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis
The most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene leads to deletion of the… 
Highly Cited
2014
Highly Cited
2014
SYNERGY-BASED SMALL-MOLECULE SCREEN USING A HUMAN LUNG EPITHELIAL CELL LINE F508-CFTR CORRECTORS THAT AUGMENT VX-809 MAXIMAL EFFICACY
The most prevalent CFTR mutation causing cystic fibrosis, Δ F508, impairs folding of nucleotide binding domain 1 (NBD1) and… 
2014
2014
Searching for Combinations of Small-Molecule Correctors to Restore F508del–Cystic Fibrosis Transmembrane Conductance Regulator Function and Processing
The mutated protein F508del–cystic fibrosis transmembrane conductance regulator (CFTR) failed to traffic properly as a result of… 
Review
2012
Review
2012
Cystic Fibrosis Transmembrane Conductance Regulator–Modifying Medications: The Future of Cystic Fibrosis Treatment
OBJECTIVE: To review and evaluate cystic fibrosis transmembrane conductance regulator (CFTR) modulators for the treatment of… 
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