title: CFTR Modulators for the Treatment of Cystic Fibrosis.
abstract: 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.
authors.forename: Rebecca S
authors.initials: RS
authors.lastname: Pettit
authors.forename: Chris
authors.initials: C
authors.lastname: Fellner
keywords: 4PBA; CFTR; CFTR correctors; CFTR modulators; CFTR potentiators; VRT-532; VX-661; ataluren; cystic fibrosis; cystic fibrosis transmembrane conductance regulator (CFTR); ivacaftor; lumacaftor
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