[HTML][HTML] CFTR modulators for the treatment of cystic fibrosis

RS Pettit, C Fellner - Pharmacy and Therapeutics, 2014 - ncbi.nlm.nih.gov
RS Pettit, C Fellner
Pharmacy and Therapeutics, 2014ncbi.nlm.nih.gov
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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.
ncbi.nlm.nih.gov