PMID: 22398597

Ledford H
Cystic fibrosis drug Vertex's latest triumph.
Nat Biotechnol. 2012 Mar 7;30(3):201-2. doi: 10.1038/nbt0312-201a., [PubMed]
Sentences
No. Mutations Sentence Comment
17 ABCC7 p.Arg117His
X
ABCC7 p.Arg117His 22398597:17:231
status: NEW
view ABCC7 p.Arg117His details
 A pediatric study will evaluate the drug in patients between two and five years old, another trial will seek to expand use of the drug in other CFTR gating mutations and a third will specifically target a class of mutations called R117H, which also affects gating but retains some degree of chloride ion transport. Login to comment 52 ABCC7 p.Gly551Asp
X
ABCC7 p.Gly551Asp 22398597:52:111
status: NEW
view ABCC7 p.Gly551Asp details
 But observers note that the decrease in sweat chloride was not as dramatic as the drop produced by Kalydeco in G551D patients. Login to comment