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Article

Genotype-dependent biochemical response to elexacaftor/tezacaftor/ivacaftor in children with cystic fibrosis: A 24-month real-world study.

Respiratory medicine - 1 Jun 2026

Simonetti Gabriele, Caporale Roberto, Carreras Giulia, Lopes-Pacheco Miquéias, Terlizzi Vito

Abstract excerpt

BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ETI) has revolutionized cystic fibrosis (CF) care; however, its efficacy in pediatric subjects carrying one ETI-responsive and one non-responsive CFTR variant remains incompletely characterized. This study compared the real-world response to ETI using sweat chloride concentration (SCC) as a key biomarker of CFTR function. METHODS: In this retrospective, single-center...

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PMID: 42067070
DOI: 10.1016/j.rmed.2026.108853

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CFTR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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