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.

SUBMITTER: Pettit RS 

PROVIDER: S-EPMC4103577 | biostudies-other | 2014 Jul

REPOSITORIES: biostudies-other