Article
Ivacaftor treatment of cystic fibrosis patients with the G551D mutation: a review of the evidence.
Therapeutic advances in respiratory disease - 1 Oct 2013
Kotha Kavitha, Clancy John P
Abstract excerpt
Cystic fibrosis (CF) is a recessive disorder caused by mutations in the gene that encodes the CF transmembrane conductance regulator (CFTR) protein. CFTR protein is a chloride and bicarbonate channel that is critical for normal epithelial ion transport and hydration of epithelial surfaces. Current CF care is supportive, but recent breakthroughs have occurred with the advent of novel therapeutic strategies that...
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