Loading...

Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation

Cystic fibrosis (CF) results from mutations in the CF transmembrane conductance regulator (CFTR) gene, which codes for the CFTR channel protein. The most common mutation in CF is F508del, which produces a misfolded protein with diminished channel activity. The development of small-molecule CFTR-modu...

Full description

Saved in:
Bibliographic Details
Published in:Expert Rev Precis Med Drug Dev
Main Authors: Cholon, Deborah M., Esther, Charles R., Gentzsch, Martina
Format: Artigo
Language:Inglês
Published: 2016
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4963025/
https://ncbi.nlm.nih.gov/pubmed/27482545
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/23808993.2016.1175299
Tags: Add Tag
No Tags, Be the first to tag this record!