Elexacaftor/tezacaftor/ivacaftor

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Elexacaftor/tezacaftor/ivacaftor (pronunciation: eh-leks-uh-KAF-tor/teh-zuh-KAF-tor/eye-vuh-KAF-tor) is a combination medication used for the treatment of cystic fibrosis in people aged six years and older who have at least one F508del mutation in the CFTR gene.

Etymology

The names of the three drugs in this combination reflect their chemical structures and mechanisms of action. The suffix '-caftor' is used for drugs that target the CFTR protein, which is dysfunctional in cystic fibrosis.

Pharmacology

Elexacaftor, tezacaftor, and ivacaftor are all modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. They work by increasing the quantity and function of the CFTR protein on the cell surface, thereby improving the transport of chloride ions across cell membranes and reducing the symptoms of cystic fibrosis.

Related Terms

  • Cystic fibrosis: A genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine.
  • CFTR: The cystic fibrosis transmembrane conductance regulator, a protein that controls the movement of chloride and thiocyanate ions across epithelial cell membranes.
  • Mutation: A change in the DNA sequence that can lead to changes in the function of the encoded protein.

See Also

External links

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