Article
Lumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR.
Drugs of today (Barcelona, Spain : 1998) - 1 Apr 2016
Zhang W, Zhang X, Zhang Y H, Strokes D C, Naren A P
Abstract excerpt
Cystic fibrosis (CF) is a life-shortening inherited disease caused by the loss or dysfunction of the CF transmembrane conductance regulator (CFTR) channel activity resulting from mutations in the CFTR gene. Phe508del is the most prevalent mutation, with approximately 90% of all CF patients carrying it on at least one allele. Over the past two or three decades, significant progress has been made in understanding...
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