VX-809, AN INVESTIGATIONAL CFTR CORRECTOR, IN COMBINATION WITH VX-770, AN INVESTIGATIONAL CFTR POTENTIATOR, IN SUBJECTS WITH CF AND HOMOZYGOUS FOR THE F508DEL-CFTR MUTATION

被引：0
|
作者：
Boyle, M. P. [1 ]
Bell, S. [2 ]
Konstan, M. W. [3 ]
McColley, S. A. [4 ]
Wisseh, S. [5 ]
Spencer-Green, G. [5 ]
机构：
[1] Johns Hopkins Univ Hosp, Baltimore, MD 21287 USA
[2] Prince Charles Hosp, Brisbane, Qld 4032, Australia
[3] Rainbow Babies & Childrens Hosp, Cleveland, OH 44106 USA
[4] Childrens Mem Hosp, Chicago, IL 60614 USA
[5] Vertex Pharmaceut, Cambridge, MA USA
来源：
PEDIATRIC PULMONOLOGY | 2011年
关键词：
D O I：
暂无
中图分类号：
R72 [儿科学];
学科分类号：
100202 ;
摘要：
引用
收藏
页码：287 / 287
页数：1
相关论文
共 50 条
  • [1] VX-770 IN SUBJECTS WITH CF AND HOMOZYGOUS FOR THE F508DEL-CFTR MUTATION
    Flume, P. A.
    Borowitz, D.
    Liou, T.
    Li, H.
    Yen, K.
    Ordonez, C.
    Geller, D. E.
    [J]. PEDIATRIC PULMONOLOGY, 2011, : 284 - 285
  • [2] 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
    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.
    [J]. THORAX, 2012, 67 (01) : 12 - 18
  • [3] Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels
    Kopeikin, Z.
    Yuksek, Z.
    Yang, H. -Y.
    Bompadre, S. G.
    [J]. JOURNAL OF CYSTIC FIBROSIS, 2014, 13 (05) : 508 - 514
  • [4] VX-770, AN INVESTIGATIONAL CFTR POTENTIATOR, ACTS ON MULTIPLE CFTR FORMS IN VITRO
    Yu, H.
    Burton, B.
    van Goor, F.
    [J]. PEDIATRIC PULMONOLOGY, 2010, : 318 - 319
  • [5] Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
    Van Goor, Fredrick
    Hadida, Sabine
    Grootenhuis, Peter D. J.
    Burton, Bill
    Stack, Jeffrey H.
    Straley, Kimberly S.
    Decker, Caroline J.
    Miller, Mark
    McCartney, Jason
    Olson, Eric R.
    Wine, Jeffrey J.
    Frizzell, Ray A.
    Ashlock, Melissa
    Negulescu, Paul A.
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2011, 108 (46) : 18843 - 18848
  • [6] LONG-TERM SAFETY AND EFFICACY OF INVESTIGATIONAL CFTR POTENTIATOR, VX-770, IN SUBJECTS WITH CF
    McKone, E. F.
    Borowitz, D.
    Drevinek, P.
    Griese, M.
    Konstan, M. W.
    Wainwright, C. E.
    Ratjen, F.
    Sermet-Gaudelus, I
    Rodriguez, S.
    Yen, K.
    Davies, J. C.
    [J]. PEDIATRIC PULMONOLOGY, 2011, : 284 - 284
  • [7] COMPARATIVE STUDY OF F508DEL-CFTR RESCUE IN RESPONSE TO COMBINATION OF CORRECTORS VX-809, SAHA, CORR4A, THE IMINOSUGARS ISOLAB AND MIGLUSTAT AND THE POTENTIATOR VX-770
    Boinot, C.
    Jollivet, M.
    Norez, C.
    Becq, F.
    [J]. PEDIATRIC PULMONOLOGY, 2013, 48 : 220 - 220
  • [8] Discovery of in situ click chemistry compatible analogs of F508del-CFTR corrector VX-809
    Brown, Olivia
    Turlington, Mark
    [J]. ABSTRACTS OF PAPERS OF THE AMERICAN CHEMICAL SOCIETY, 2019, 257
  • [9] VX-809 AND VX-770 TREATMENT IN RECTAL CF ORGANOIDS EXPRESSING UNCOMMON CFTR GENOTYPES
    Dekkers, F.
    Kruisselbrink, E.
    de Jonge, H.
    Bronsveld, I
    de Winter, K.
    Janssens, H.
    Clevers, H.
    van der Ent, K.
    Vijftigschild, L.
    [J]. PEDIATRIC PULMONOLOGY, 2013, 48 : 269 - 269
  • [10] THE INVESTIGATIONAL CFTR CORRECTOR, VX-809 (LUMACAFTOR) CO-ADMINISTERED WITH THE ORAL POTENTIATOR IVACAFTOR IMPROVED CFTR AND LUNG FUNCTION IN F508DEL HOMOZYGOUS PATIENTS: PHASE II STUDY RESULTS
    Boyle, M. P.
    Bell, S.
    Konstan, M.
    McColley, S. A.
    Kang, L.
    Patel, N.
    [J]. PEDIATRIC PULMONOLOGY, 2012, 47 : 315 - 315
12345