Article
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial.
The Lancet. Respiratory medicine - 1 Jul 2017
Ratjen Felix, Hug Christopher, Marigowda Gautham, Tian Simon, Huang Xiaohong, Stanojevic Sanja, Milla Carlos E, Robinson Paul D, Waltz David, Davies Jane C
Abstract excerpt
BACKGROUND: Lumacaftor and ivacaftor combination treatment showed efficacy in patients aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis transmembrane conductance regulator (CFTR) in placebo-controlled studies and patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR in an open-label study. We report efficacy and safety of lumacaftor and ivacaftor in patients...
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