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

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 <i>Phe508del</i>/unknown CFTR genotype. An ex vivo analysis on nasal epithelial cells (NEC) indicated a significant improvement of CFTR gating activity after the tre...

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Bibliographic Details
Main Authors: Vito Terlizzi, Carmela Colangelo, Giovanni Marsicovetere, Michele D’Andria, Michela Francalanci, Diletta Innocenti, Eleonora Masi, Angelo Avarello, Giovanni Taccetti, Felice Amato, Marika Comegna, Giuseppe Castaldo, Donatello Salvatore
Format: Article
Language:English
Published: MDPI AG 2021-07-01
Series:Genes
Subjects:
elexacaftor/tezacaftor/ivacaftor
cystic fibrosis
CFTR
nasal brushing
sweat chloride
Online Access:https://www.mdpi.com/2073-4425/12/8/1178

Internet

https://www.mdpi.com/2073-4425/12/8/1178

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