Successful Therapy over 12 Months of People with Cystic Fibrosis with Rare Non-phe508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutations with Elexacaftor/Tezacaftor/Ivacaftor (ETI)

Successful Therapy over 12 Months of People with Cystic Fibrosis with Rare Non-phe508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutations with Elexacaftor/Tezacaftor/Ivacaftor (ETI)

<b>Background:</b> Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a CFTR modulator therapy approved for people with cystic fibrosis (pwCF) who have at least one phe508del mutation. However, its approval in the European Union (EU) for pwCF with non-phe508del mutations is lacking, because data...

Full description

Bibliographic Details
Main Authors: Tomke Sütering, Sebastian F. N. Bode, Rainald Fischer, Dorit Fabricius
Format: Article
Language:English
Published: MDPI AG 2024-12-01
Series:Advances in Respiratory Medicine
Subjects:
elexacaftor/tezacaftor/ivacaftor
cystic fibrosis
rare CFTR mutations
non-phe508del CFTR mutations
off-label use
Online Access:https://www.mdpi.com/2543-6031/92/6/49

Internet

https://www.mdpi.com/2543-6031/92/6/49

Similar Items