Article
Ivacaftor: the first therapy acting on the primary cause of cystic fibrosis.
Drugs of today (Barcelona, Spain : 1998) - 1 Apr 2013
McPhail G L, Clancy J P
Abstract excerpt
Cystic fibrosis (CF) is a life-shortening disorder that affects over 30,000 people in the U.S. and 70,000 worldwide. CF is caused by mutations in the CFTR gene, which codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. CFTR is a chloride and bicarbonate channel and regulates several ion transporters at the epithelial cell membrane, controlling hydration or ionic composition of...
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