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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Detalles Bibliográficos
Autores principales: Leung, Winnie M., Davoodi, Parastoo Molla, Langevin, Ashten, Smith, Clare, Parkins, Michael D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10618485/
https://www.ncbi.nlm.nih.gov/pubmed/37920361
http://dx.doi.org/10.1016/j.rmcr.2023.101938

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