Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are a class of drugs that act by improving production, intracellular processing, and/or function of the defective CFTR protein.
How do CFTR correctors work?
Corrector CFTR modulators, like lumacaftor or tezacaftor, attempt to stabilize the CFTR protein so it can work properly. They help to fix the folding process so that the puzzle pieces of the protein fit together. When the CFTR protein is in the right shape, it is able to move to the cell surface.
What is modulator therapy?
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies are designed to correct the malfunctioning protein made by the CFTR gene.