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Elexacaftor: next-generation modulator of the CFTR protein

Publication at Second Faculty of Medicine |
2021

Abstract

Causal treatment of cystic fibrosis became a clinical reality in 2012 thanks to the CFTR protein (cystic fibrosis transmembrane conductance regulator) modulators. This year, the range of these drugs in the Czech Republic has expanded by a combination of elexacaftor, tezacaftor and ivacaftor for carriers of at least one F508del mutation of the CFTR gene at the age of at least 12 years.

If the use is extended to younger age categories, causal treatment will be available for 90% of CF patients in the Czech Republic.