ABCMdb

PMID: 21372122

Narasimhan M, Cohen R
New and investigational treatments in cystic fibrosis.
Ther Adv Respir Dis. 2011 Aug;5(4):275-82. Epub 2011 Mar 3., [PubMed]

Sentences

No. Mutations Sentence Comment

145 ABCC7 p.Gly551Asp ABCC7 p.Arg117His VX-770 (Vertex pharmaceuticals) is a compound that increases cyclic-AMP-dependent chloride secretion in cell cultures, and is a potent and selective CFTR potentiator of wild-type, G551D, F508del and R117H forms of CFTR. Login to comment 146 ABCC7 p.Gly551Asp ABCC7 p.Gly551Asp ABCC7 p.Arg117His In a small phase 2 clinical trial, VX-770 administered twice daily in 39 patients with the G551D mutation over a 14-day or 28-day period improved lung function, nasal potential difference measures, and sweat chloride levels [Accurso et al. 2010]. Login to comment 147 ABCC7 p.Gly551Asp In a small phase 2 clinical trial, VX-770 administered twice daily in 39 patients with the G551D mutation over a 14-day or 28-day period improved lung function, nasal potential difference measures, and sweat chloride levels [Accurso et al. 2010]. Login to comment