Article
CFTR Modulators: Current Status and Evolving Knowledge.
Seminars in respiratory and critical care medicine - 1 Apr 2023
Regard Lucile, Martin Clémence, Da Silva Jennifer, Burgel Pierre-Régis
Abstract excerpt
In the past decade, the medical management of people with cystic fibrosis (pwCF) has changed with the development of small molecules that partially restore the function of the defective CF transmembrane conductance regulator (CFTR) protein and are called CFTR modulators. Ivacaftor (IVA), a CFTR potentiator with a large effect on epithelial ion transport, was the first modulator approved in pwCF carrying gating...
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