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

Valentina FainardiFederico CrestaClaudio SorioPaola MelottiEmanuela PesceMichela DeolmiFrancesco LongoKleinfelder KarinaSusanna EspositoGiovanna Pisi

Published in: Pediatric pulmonology (2024)

Despite the limited number of cases, our results support the use of CFTR modulators in patients with rare CFTR variants that are not currently approved for ETI in Europe.

Keyphrases

cystic fibrosis
small molecule
copy number
drug administration
dna methylation
genome wide