Abstract
CFTR modulators have been available since 2012 for causal treatment of cystic fibrosis. These are substances that affect specific mutations or groups of mutations of the CFTR gene.
In general, they can be divided into CFTR correctors and potentiators, where CFTR correctors increase the amount of CFTR protein in the cell membrane, while CFTR potentiators improve its function. In clinical practice, the CFTR potentiator ivacaftor and the CFTR correctors lumacaftor and tezacaftor are currently used.
Another CFTR corrector, elexacaftor, is expected to be approved for clinical use in the near future.