Article
VOCAL: An observational study of ivacaftor for people with cystic fibrosis and selected non-G551D-CFTR gating mutations.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2023
Simmonds Nicholas J, van der Ent C Kors, Colombo Carla, Kinnman Nils, DeSouza Cynthia, Thorat Teja, Chew Marci L, Chandarana Keval, Castellani Carlo
Abstract excerpt
BACKGROUND: VOCAL was an observational study of the effect of long-term ivacaftor on real-world clinical outcomes and healthcare resource utilization (HCRU) in people with cystic fibrosis (pwCF) in Italy, the Netherlands, and the UK. METHODS: pwCF aged ≥6 years with non-G551D-CFTR gating mutations were eligible. Prospective data were collected up to 48 months after enrollment; retrospective data were collected to...
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