Elexacaftor-Tezacaftor-Ivacaftor in 2 cystic fibrosis adults homozygous for M1101K with end-stage lung disease

Elexacaftor-tezacaftor-ivacaftor (ETI) therapy is shown to improve the health of individuals with cystic fibrosis (CF) who have the F508del variant. There are in vitro studies showing benefit with ETI for select rare CF variants. Limited data exists on the use of ETI in individuals with rare CF vari...

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Principais autores:	Winnie M. Leung, Parastoo Molla Davoodi, Ashten Langevin, Clare Smith, Michael D. Parkins
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2023-01-01
coleção:	Respiratory Medicine Case Reports
Assuntos:	Cystic fibrosis Cystic fibrosis transmembrane conductance regulator CFTR modulator Elexacaftor-tezacaftor-ivacaftor
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2213007123001338
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