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

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.

KEYWORDS:

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

PMID:
25083129
PMCID:
PMC4103577

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