1. 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. Issue 9 (September 2021) Authors: Hoppe, Jordana E; Chilvers, Mark; Ratjen, Felix; McNamara, John J; Owen, Caroline A; Tian, Simon; Zahigian, Rachel; Cornell, Alexandra G; McColley, Susanna A Journal: Lancet Issue: Volume 9:Issue 9(2021) Page Start: 977 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. Issue 4 (April 2019) Authors: McNamara, John J; McColley, Susanna A; Marigowda, Gautham; Liu, Fang; Tian, Simon; Owen, Caroline A; Stiles, David; Li, Chonghua; Waltz, David; Wang, Linda T; Sawicki, Gregory S Journal: Lancet Issue: Volume 7:Issue 4(2019) Page Start: 325 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗