Article
Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study.
The Lancet. Respiratory medicine - 1 Sept 2021
Hoppe Jordana E, Chilvers Mark, Ratjen Felix, McNamara John J, Owen Caroline A, Tian Simon, Zahigian Rachel, Cornell Alexandra G, McColley Susanna A
Abstract excerpt
BACKGROUND: A previous phase 3 study showed that lumacaftor-ivacaftor was generally safe and well tolerated over 24 weeks of treatment in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation. In this study, we aimed to assess the long-term safety of lumacaftor-ivacaftor in a rollover study of children who participated in this previous phase 3 study. METHODS: In this multicentre,...
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