Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation
- Davies, Jane C.
- Wainwright, Claire E.
- Canny, Gerard J.
- Chilvers, Mark A.
- Howenstine, Michelle S.
- Munck, Anne
- Mainz, Jochen G.
- Rodriguez, Sally
- Li, Haihong
- Yen, Karl
- Ordonez, Claudia L.
- Ahrens, Richard
Publication date
June 2013
DOI Publisher
American Thoracic Society ISSN
1073-449X Citation count (estimate)
220Abstract
Lumacaftor/ivacaftor combination therapy has shown clinical benefits in patients with cystic fibrosis homozygous for the Phe508del CFTR mutation; however, pretreatment lung function is a confounding factor that potentially affects the efficacy and safety of this therapy. We aimed to assess the efficacy and safety of lumacaftor/ivacaftor therapy in these patients, defined by specific categories of lung function
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