CFTR Modulators for the Treatment of Cystic Fibrosis

P T. 2014 Jul;39(7):500-11.


Defects in a single gene lead to the defective proteins that cause cystic fibrosis, making the disease an ideal candidate for mutation-targeted therapy. Although ivacaftor is currently the only FDA-approved CFTR modifier, others are in development.

Keywords: 4PBA; CFTR; CFTR correctors; CFTR modulators; CFTR potentiators; VRT-532; VX-661; ataluren; cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); ivacaftor; lumacaftor.