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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

Semantic Scholar uses AI to extract papers important to this topic.
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… 
2017
2017
Complexity of phenotypes induced by p.Asn1303Lys-CFTR correlates with difficulty to rescue and activate this protein.
Cystic Fibrosis is the most common recessive autosomal rare disease found in Caucasian. It is caused by mutations on the Cystic… 
2016
2016
A synonymous codon change alters the drug sensitivity of ΔF508 cystic fibrosis transmembrane conductance regulator
Synonymous mutations, such as I507‐ATCÅATT, in deletion of Phe508 in cystic fibrosis transmembrane conductance regulator (ΔF508… 
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… 
2016
2016
An Investigational Anti‐Cancer Agent Synergizes with VX‐809 in F508del Correction
Cystic Fibrosis (CF) is caused by functional deficiency of cystic fibrosis transmembrane conductance regulator (CFTR), an anion… 
2015
2015
A milestone in the investigation of population-wide effects of vaccines.
Development Network. Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in… 
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… 
2013
2013
New cystic fibrosis drug paves the way for orphan diseases.
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… 
2012
2012
Lumacaftor (VX-809)
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