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A retrospective analysis of clinical parameters, safety and tolerability of Elexacaftor/Tezacaftor/Ivacaftor in a cohort of patients aged 6-17 years with cystic fibrosis

2025-01-15

Abstract excerpt

<h4>Background: </h4> Elexacaftor/Tezacaftor/Ivacaftor (ETI) improved clinical outcomes in patients with cystic fibrosis having at least one copy of the Phe508del CFTR mutation [1]. However, ETI safety and efficacy in pediatric setting is poorly documented. <h4>Methods: </h4> A retrospective, single-center study of 27 patients, aged 6-17 years, followed for at least 6 months between October 2022 and March 2024, w...

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Literature Corpus work
fa200aad-603c-5c65-9583-f9553e4d2498
DOI
10.22541/au.173697416.66632785/v1
Open publication

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