Article
Advancing clinical development pathways for new CFTR modulators in cystic fibrosis.
Thorax - 1 May 2016
Mayer-Hamblett Nicole, Boyle Michael, VanDevanter Donald
Abstract excerpt
Cystic fibrosis (CF) is a life-shortening genetic disease affecting approximately 70,000 individuals worldwide. Until recently, drug development efforts have emphasised therapies treating downstream signs and symptoms resulting from the underlying CF biological defect: reduced function of the CF transmembrane conductance regulator (CFTR) protein. The current CF drug development landscape has expanded to include...
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