Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor Therapy in Three Subjects with the Cystic Fibrosis Genotype Phe508del/Unknown and Advanced Lung Disease

We evaluated the effectiveness and safety of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in three subjects carrying the Phe508del/unknown CFTR genotype. An ex vivo analysis on nasal epithelial cells (NEC) indicated a significant improvement of CFTR gating activity after the treatment. Three patie...

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Detalles Bibliográficos
Autores principales: Terlizzi, Vito, Colangelo, Carmela, Marsicovetere, Giovanni, D’Andria, Michele, Francalanci, Michela, Innocenti, Diletta, Masi, Eleonora, Avarello, Angelo, Taccetti, Giovanni, Amato, Felice, Comegna, Marika, Castaldo, Giuseppe, Salvatore, Donatello
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8391133/
https://www.ncbi.nlm.nih.gov/pubmed/34440351
http://dx.doi.org/10.3390/genes12081178

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