Article
Impact of the expanded label for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with no F508del variant in the USA.
The European respiratory journal - 1 Nov 2024
Cromwell Elizabeth A, Ostrenga Josh S, Sanders Don B, Morgan Wayne, Castellani Carlo, Szczesniak Rhonda, Burgel Pierre-Regis
Abstract excerpt
BACKGROUND: Elexacaftor/tezacaftor/ivacaftor (ETI), which is approved for people with cystic fibrosis (pwCF) with a F508del variant, was further approved based on in vitro data in the USA for those carrying at least one of 177 rare CFTR (cystic fibrosis transmembrane conductance regulator) variants. METHODS: PwCF, aged ≥6 years, carrying no F508del variant but with at least one of these 177 rare variants, were...
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