Article
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.
The Lancet. Respiratory medicine - 1 Feb 2017
Konstan Michael W, McKone Edward F, Moss Richard B, Marigowda Gautham, Tian Simon, Waltz David, Huang Xiaohong, Lubarsky Barry, Rubin Jaime, Millar Stefanie J, Pasta David J, Mayer-Hamblett Nicole, Goss Christopher H, Morgan Wayne, Sawicki Gregory S
Abstract excerpt
BACKGROUND: The 24-week safety and efficacy of lumacaftor/ivacaftor combination therapy was shown in two randomised controlled trials (RCTs)-TRAFFIC and TRANSPORT-in patients with cystic fibrosis who were aged 12 years or older and homozygous for the F508del-CFTR mutation. We aimed to assess the long-term safety and efficacy of extended lumacaftor/ivacaftor therapy in this group of patients in PROGRESS, the...
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