Article
Elexacaftor/tezacaftor/ivacaftor projected survival and long-term health outcomes in people with cystic fibrosis homozygous for F508del.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jul 2023
Lopez Andrea, Daly Conor, Vega-Hernandez Gabriela, MacGregor Gordon, Rubin Jaime L
Abstract excerpt
BACKGROUND: A series of phase 3 clinical trials have demonstrated that elexacaftor plus tezacaftor plus ivacaftor (ELX/TEZ/IVA) is safe and efficacious in people with cystic fibrosis (pwCF) aged ≥12 years with ≥1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The impact of this treatment on lifetime clinical outcomes and survival, however, has yet to be assessed. METHODS:...
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