Ivacaftor in a G551D Homozygote with Cystic Fibrosis
Volume: 369, Issue: 13, Pages: 1280 - 1282
Published: Sep 26, 2013
Abstract
Ivacaftor, an oral potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, has been shown to be efficacious in patients heterozygous for the G551D mutation in CFTR. This letter describes the effect of ivacaftor in a patient homozygous for this...
Paper Details
Title
Ivacaftor in a G551D Homozygote with Cystic Fibrosis
Published Date
Sep 26, 2013
Volume
369
Issue
13
Pages
1280 - 1282
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