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 surface with...

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Hlavní autoři:	Francesca Lucca, Sonia Volpi, Mirco Ros, Benedetta Fabrizzi, Ilaria Meneghelli, Marica Bordicchia, Francesca Buniotto, Alessia Lancini, Cecilia Brignole, Francesca Pauro, Valentino Bezzerri, Marco Cipolli
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	MDPI AG 2025-03-01
Edice:	International Journal of Translational Medicine
Témata:	elexacaftor/tezacaftor/ivacaftor CFTR modulators cystic fibrosis N1303K off-label use
On-line přístup:	https://www.mdpi.com/2673-8937/5/1/11
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