Efficacy of elexacaftor/tezacaftor/ivacaftor in non-cystic fibrosis bronchiectasis patients with a single pathogenic CFTR mutation: a retrospective cohort analysis

Abstract excerpt

Introduction It has previously been established that there is an increased incidence of mutations in the cystic fibrosis transmembrane regulator ( CFTR ) gene amongst patients with non-cystic fibrosis bronchiectasis (NCFB). With the advent of new highly effective CFTR modulators, specifically elexacaftor–tezacaftor–ivacaftor (ETI), we asked whether NCFB patients with a single ETI amenable mutation who do not meet...

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