Long-term clinical outcomes of elexacaftor/tezacaftor/ivacaftor therapy in adults with cystic fibrosis and advanced pulmonary disease

The combination of cystic fibrosis transmembrane conductance regulator (CFTR) modulators elexacaftor, tezacaftor and ivacaftor (ELX/TEZ/IVA) has been approved for treatment of cystic fibrosis (CF) patients (pwCF) homozygous and heterozygous for Phe508del. We aim to assess the long-term effects of ELX/TEZ/IVA therapy on clinical outcomes in severe pwCF.

Source: Respiratory Medicine - September 8, 2023 Category: Respiratory Medicine Authors: Daniela Savi, Francesca Lucca, Gloria Tridello, Ilaria Meneghelli, Isabella Comello, Sara Tomezzoli, Monica Signorini, Elena Proietti, Giulia Cucchetto, Sonia Volpi, Marco Cipolli Tags: Original Research Source Type: research