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

This study, which included 57 of 60 participants who received lumacaftor–ivacaftor in the 24-week Phase III study, found side-effects were mainly mild or moderate (most common included cough and nasal congestion), and treatment effects were generally maintained up to 120 weeks.

The Lancet Respiratory Medicine