Article
Optimizing nasal potential difference analysis for CFTR modulator development: assessment of ivacaftor in CF subjects with the G551D-CFTR mutation.
PloS one - 1 Jan 2013
Rowe Steven M, Liu Bo, Hill Aubrey, Hathorne Heather, Cohen Morty, Beamer John R, Accurso Frank J, Dong Qunming, Ordoñez Claudia L, Stone Anne J, Olson Eric R, Clancy John P
Abstract excerpt
Nasal potential difference (NPD) is used as a biomarker of the cystic fibrosis transmembrane conductance regulator (CFTR) and epithelial sodium channel (ENaC) activity. We evaluated methods to detect changes in chloride and sodium transport by NPD based on a secondary analysis of a Phase II CFTR-modulator study. Thirty-nine subjects with CF who also had the G551D-CFTR mutation were randomized to receive ivacaftor...
Topics
- Adult
- Amino Acid Substitution
- Aminophenols
- Biological Transport
- Chlorides
- Cystic Fibrosis
- Cystic Fibrosis Transmembrane Conductance Regulator
- Demography
- Epithelial Sodium Channels