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 on treatment...

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Auteurs principaux:	Tomke Sütering, Sebastian F. N. Bode, Rainald Fischer, Dorit Fabricius
Format:	Artigo
Langue:	Inglês
Publié:	MDPI AG 2024-12-01
Collection:	Advances in Respiratory Medicine
Sujets:	elexacaftor/tezacaftor/ivacaftor cystic fibrosis rare CFTR mutations non-phe508del CFTR mutations off-label use
Accès en ligne:	https://www.mdpi.com/2543-6031/92/6/49
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