Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two F508del Alleles.

doi:10.1164/rccm.202110-2249OC

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Article

Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two F508del Alleles.

American journal of respiratory and critical care medicine - 1 Mar 2022

Graeber Simon Y, Vitzthum Constanze, Pallenberg Sophia T, Naehrlich Lutz, Stahl Mirjam, Rohrbach Alexander, Drescher Marika, Minso Rebecca, Ringshausen Felix C, Rueckes-Nilges Claudia, Klajda Jan, Berges Julian, Yu Yin, Scheuermann Heike, Hirtz Stephanie, Sommerburg Olaf, Dittrich Anna-Maria, Tümmler Burkhard, Mall Marcus A

Abstract excerpt

Rationale: The CFTR (cystic fibrosis transmembrane conductance regulator) modulator combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical outcomes and sweat chloride concentration in patients with cystic fibrosis (CF) and one or two F508del alleles. However, the effect of ELX/TEZ/IVA on CFTR function in the airways and intestine has not been studied. Objectives: To assess the...

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PMID: 34936849
DOI: 10.1164/rccm.202110-2249OC

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