Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in children with cystic fibrosis aged 2-5 years (TIMBERLINE Trial VX21-121-105): a phase 3, open-label study.
BACKGROUND Vanzacaftor-tezacaftor-deutivacaftor was shown to be efficacious and safe in people with cystic fibrosis aged 6 years and older, providing further restoration of CFTR function compared with elexacaftor-tezacaftor-ivacaftor. We aimed to assess the safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in...