VX-770 IN SUBJECTS 6 TO 11 YEARS WITH CYSTIC FIBROSIS AND THE G551D-CFTR MUTATION

被引:0
|
作者
Aherns, R. [1 ]
Rodriguez, S. [2 ]
Yen, K. [2 ]
Davies, J. C. [3 ]
机构
[1] Univ Iowa, Iowa City, IA USA
[2] Vertex Pharmaceut Inc, Cambridge, MA USA
[3] Royal Brompton Hosp, London SW3 6LY, England
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:283 / 283
页数:1
相关论文
共 50 条
  • [21] Pseudomonas aeruginosa adaptions in cystic fibrosis patients with G551D-CFTR mutations treated with ivacaftor
    Zhou, H.
    Smalley, N. E.
    Xu, F.
    Dandekar, A. A.
    EUROPEAN JOURNAL OF IMMUNOLOGY, 2019, 49 : 1335 - 1335
  • [22] 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
    AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2013, 187 (11) : 1219 - 1225
  • [23] THE EFFECT OF IVACAFTOR ON WEIGHT OVER THREE YEARS IN PATIENTS WITH CF AND A G551D-CFTR MUTATION
    Sawicki, G. S.
    McKone, E. F.
    Pasta, D. J.
    Wagener, J.
    Johnson, C.
    Millar, S. J.
    Konstan, M.
    PEDIATRIC PULMONOLOGY, 2014, 49 : 289 - 289
  • [24] 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
    Boyle, M. P.
    Bell, S.
    Konstan, M. W.
    McColley, S. A.
    Wisseh, S.
    Spencer-Green, G.
    PEDIATRIC PULMONOLOGY, 2011, : 287 - 287
  • [25] Revertant mutants modify, but do not rescue, the gating defect of the cystic fibrosis mutant G551D-CFTR
    Xu, Zhe
    Pissarra, Luisa S.
    Farinha, Carlos M.
    Liu, Jia
    Cai, Zhiwei
    Thibodeau, Patrick H.
    Amaral, Margarida D.
    Sheppard, David N.
    JOURNAL OF PHYSIOLOGY-LONDON, 2014, 592 (09): : 1931 - 1947
  • [26] Pharmacokinetics (PK), Metabolism and Excretion of [14C] VX-770, a Potentiator of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), in Healthy Subjects
    Zha, Jiuhong
    Zhang, Jianbo
    Ordonez, Claudia
    JOURNAL OF CLINICAL PHARMACOLOGY, 2011, 51 (09): : 1358 - 1359
  • [27] Direct interaction of a small-molecule modulator with G551D-CFTR, a cystic fibrosis-causing mutation associated with severe disease
    Pasyk, Stan
    Li, Canhui
    Ramjeesingh, Mohabir
    Bear, Christine E.
    BIOCHEMICAL JOURNAL, 2009, 418 : 185 - 190
  • [28] A small molecule CFTR potentiator restores ATP-dependent channel gating to the cystic fibrosis mutant G551D-CFTR
    Liu, Jia
    Berg, Allison P.
    Wang, Yiting
    Jantarajit, Walailak
    Sutcliffe, Katy J.
    Stevens, Edward B.
    Cao, Lishuang
    Pregel, Marko J.
    Sheppard, David N.
    BRITISH JOURNAL OF PHARMACOLOGY, 2022, 179 (07) : 1319 - 1337
  • [29] Heterogeneity of phenotype in two cystic fibrosis patients homozygous for the CFTR exon 11 mutation G551D
    Parad, RB
    JOURNAL OF MEDICAL GENETICS, 1996, 33 (08) : 711 - 713
  • [30] Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States
    Taylor-Cousar, Jennifer
    Niknian, Minoo
    Gilmartin, Geoffrey
    Pilewski, Joseph M.
    JOURNAL OF CYSTIC FIBROSIS, 2016, 15 (01) : 116 - 122