Breakthrough therapy targets defective protein
Silver Spring, Maryland–(ENEWSPF)–January 31, 2012. The U.S. Food and Drug Administration today approved Kalydeco (ivacaftor) for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.
CF is a serious genetic disorder affecting the lungs and other organs that ultimately leads to an early death. It is caused by…
