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A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation
Author(s) -
Bonnie W. Ramsey,
Jane C. Davies,
Noel G. McElvaney,
Elizabeth Tullis,
Scott C. Bell,
Pavel Dřevínek,
Matthias Griese,
Edward F. McKone,
Claire Wainwright,
Michael W. Konstan,
Richard B. Moss,
Félix Ratjen,
Isabelle SermetGaudelus,
Steven M. Rowe,
Qunming Dong,
Sally Rodriguez,
Karl Yen,
Claudia L. Ordoñez,
J.S. Elborn
Publication year - 2011
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1105185
Subject(s) - potentiator , cystic fibrosis , medicine , ivacaftor , cystic fibrosis transmembrane conductance regulator , mutation , genetics , pharmacology , biology , gene
Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis.

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