Ivacaftor

被引:68
作者
Davis, Pamela B. [1 ]
Yasothan, Uma [2 ]
Kirkpatrick, Peter
机构
[1] Case Western Reserve Univ, Sch Med, Cleveland, OH 44106 USA
[2] IMS Hlth, London NW1 6JB, England
关键词
CYSTIC-FIBROSIS; CFTR POTENTIATOR; MUTATION;
D O I
10.1038/nrd3723
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
First disease-modifying drug approved in the United States for the treatment of cystic fibrosis in patients with a specific mutation in the gene encoding the cystic fibrosis transmembrane conductance regulator.
引用
收藏
页码:349 / 350
页数:2
相关论文
共 7 条
[1]   Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation [J].
Clancy, J. P. ;
Rowe, Steven M. ;
Accurso, Frank J. ;
Aitken, Moira L. ;
Amin, Raouf S. ;
Ashlock, Melissa A. ;
Ballmann, Manfred ;
Boyle, Michael P. ;
Bronsveld, Inez ;
Campbell, Preston W. ;
De Boeck, Kris ;
Donaldson, Scott H. ;
Dorkin, Henry L. ;
Dunitz, Jordan M. ;
Durie, Peter R. ;
Jain, Manu ;
Leonard, Anissa ;
Mccoy, Karen S. ;
Moss, Richard B. ;
Pilewski, Joseph M. ;
Rosenbluth, Daniel B. ;
Rubenstein, Ronald C. ;
Schechter, Michael S. ;
Botfield, Martyn ;
Ordonez, Claudia L. ;
Spencer-Green, George T. ;
Vernillet, Laurent ;
Wisseh, Steve ;
Yen, Karl ;
Konstan, Michael W. .
THORAX, 2012, 67 (01) :12-18
[2]   Cystic fibrosis [J].
O'Sullivan, Brian P. ;
Freedman, Steven D. .
LANCET, 2009, 373 (9678) :1891-1904
[3]   A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation [J].
Ramsey, Bonnie W. ;
Davies, Jane ;
McElvaney, N. Gerard ;
Tullis, Elizabeth ;
Bell, Scott C. ;
Drevinek, Pavel ;
Griese, Matthias ;
McKone, Edward F. ;
Wainwright, Claire E. ;
Konstan, Michael W. ;
Moss, Richard ;
Ratjen, Felix ;
Sermet-Gaudelus, Isabelle ;
Rowe, Steven M. ;
Dong, Qunming ;
Rodriguez, Sally ;
Yen, Karl ;
Ordonez, Claudia ;
Elborn, J. Stuart .
NEW ENGLAND JOURNAL OF MEDICINE, 2011, 365 (18) :1663-1672
[4]   Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis [J].
Sermet-Gaudelus, Isabelle ;
De Boeck, Kris ;
Casimir, Georges J. ;
Vermeulen, Francois ;
Leal, Teresinha ;
Mogenet, Agnes ;
Roussel, Delphine ;
Fritsch, Janine ;
Hanssens, Laurence ;
Hirawat, Samit ;
Miller, Nilsen L. ;
Constantine, Scott ;
Reha, Allen ;
Ajayi, Temitayo ;
Elfring, Gary L. ;
Miller, Langdon L. .
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2010, 182 (10) :1262-1272
[5]  
US Food and Drug Administration, 2012, FDA LAB INF KAL
[6]   Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770 [J].
Van Goor, Fredrick ;
Hadida, Sabine ;
Grootenhuis, Peter D. J. ;
Burton, Bill ;
Cao, Dong ;
Neuberger, Tim ;
Turnbull, Amanda ;
Singh, Ashvani ;
Joubran, John ;
Hazlewood, Anna ;
Zhou, Jinglan ;
McCartney, Jason ;
Arumugam, Vijayalaksmi ;
Decker, Caroline ;
Yang, Jennifer ;
Young, Chris ;
Olson, Eric R. ;
Wine, Jeffery J. ;
Frizzell, Raymond A. ;
Ashlock, Melissa ;
Negulescu, Paul .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2009, 106 (44) :18825-18830
[7]   Ivacaftor potentiation of multiple CFTR channels with gating mutations [J].
Yu, Haihui ;
Burton, Bill ;
Huang, Chien-Jung ;
Worley, Jennings ;
Cao, Dong ;
Johnson, James P., Jr. ;
Urrutia, Art ;
Joubran, John ;
Seepersaud, Sheila ;
Sussky, Katherine ;
Hoffman, Beth J. ;
Van Goor, Fredrick .
JOURNAL OF CYSTIC FIBROSIS, 2012, 11 (03) :237-245