Elexacaftor/Tezacaftor/Ivacaftor Efficacy in a Cohort of Italian Patients with CFTR Rare Mutations

Elexacaftor/Tezacaftor/Ivacaftor Efficacy in a Cohort of Italian Patients with CFTR Rare Mutations

Background: Cystic Fibrosis is an inherited disorder caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (<i>CFTR</i>) gene, encoding a chloride and bicarbonate channel widely expressed in epithelia. Loss of CFTR function leads to dehydration of the epithelium...

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Main Authors: Francesca Lucca, Sonia Volpi, Mirco Ros, Benedetta Fabrizzi, Ilaria Meneghelli, Marica Bordicchia, Francesca Buniotto, Alessia Lancini, Cecilia Brignole, Francesca Pauro, Valentino Bezzerri, Marco Cipolli
Format: Article
Language:English
Published: MDPI AG 2025-03-01
Series:International Journal of Translational Medicine
Subjects:
elexacaftor/tezacaftor/ivacaftor
CFTR modulators
cystic fibrosis
N1303K
off-label use
Online Access:https://www.mdpi.com/2673-8937/5/1/11
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