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Elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and rare mutations.

Pediatric pulmonology - 1 Dec 2024

Fainardi Valentina, Cresta Federico, Sorio Claudio, Melotti Paola, Pesce Emanuela, Deolmi Michela, Longo Francesco, Karina Kleinfelder, Esposito Susanna, Pisi Giovanna

Abstract excerpt

INTRODUCTION: The triple combination of elexacaftor/tezacaftor/ivacaftor (ETI) has dramatically improved the outcome of people with Cystic Fibrosis (pwCF) with at least one F508del mutation. However, carriers of rare cystic fibrosis transmembrane conductance regulator (CFTR) variants are not candidates for this innovative treatment. METHODS: In this observational study, we report the results of the compassionate...

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PMID: 39212240
DOI: 10.1002/ppul.27211

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CFTR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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