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. and Ahrens, Richard (2013) Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation. American Journal of Respiratory and Critical Care Medicine, 187 11: 1219-1225. doi:10.1164/rccm.201301-0153OC


Author 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
Title Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation
Journal name American Journal of Respiratory and Critical Care Medicine   Check publisher's open access policy
ISSN 1073-449X
1535-4970
Publication date 2013-06-01
Year available 2013
Sub-type Article (original research)
DOI 10.1164/rccm.201301-0153OC
Open Access Status Not Open Access
Volume 187
Issue 11
Start page 1219
End page 1225
Total pages 7
Place of publication New York, NY, U.S.A.
Publisher American Thoracic Society
Language eng
Abstract 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.
Keyword cystic fibrosis
cystic fibrosis transmembrane conductance regulator protein
cystic fibrosis
pulmonary
G551D-CFTR mutation
sweat test
Pulmonary-Function
Cftr Potentiator
Growth
Children
Disease
Identification
Pathogenesis
Vx-770
Gene
Q-Index Code C1
Q-Index Status Confirmed Code
Institutional Status UQ

 
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