RETROSPECTIVE ANALYSIS OF PHYSIOLOGICAL RESPONSE PATTERNS TO TEZACAFTOR/IVACAFTOR IN PATIENTS WITH CYSTIC FIBROSIS HOMOZYGOUS FOR F508DEL-CFTR OR HETEROZYGOUS FOR F508DEL-CFTR AND A RESIDUAL FUNCTION MUTATION

被引：1
|
作者：
Ingenito, E. [1 ]
Nair, N. [1 ]
Yi, B. [1 ]
Lekstrom-Himes, J. [1 ]
Elborn, J. S. [2 ,3 ]
Rowe, S. M. [4 ]
机构：
[1] Vertex Pharmaceut Inc, Boston, MA USA
[2] Imperial Coll, London, England
[3] Royal Brompton Hosp, London, England
[4] Univ Alabama Birmingham, Birmingham, AL USA
来源：
THORAX | 2018年 / 73卷
关键词：
D O I：
10.1136/thorax-2018-212555.75
中图分类号：
R56 [呼吸系及胸部疾病];
学科分类号：
摘要：
S69
引用
收藏
页码：A42 / A43
页数：2
相关论文
共 50 条
  • [1] RETROSPECTIVE ANALYSIS OF PHYSIOLOGICAL RESPONSE PATTERNS TO TEZACAFTOR/IVACAFTOR IN PATIENTS WITH CYSTIC FIBROSIS HOMOZYGOUS FOR F508DEL-CFTR OR HETEROZYGOUS FOR F508DEL-CFTR AND A RESIDUAL FUNCTION MUTATION
    Ingenito, E.
    Nair, N.
    Yi, B.
    Lekstrom-Himes, J.
    Elborn, J.
    Rowe, S. M.
    [J]. PEDIATRIC PULMONOLOGY, 2018, 53 : 198 - 198
  • [2] Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
    Flume, Patrick A.
    Liou, Theodore G.
    Borowitz, Drucy S.
    Li, Haihong
    Yen, Karl
    Ordonez, Claudia L.
    Geller, David E.
    [J]. CHEST, 2012, 142 (03) : 718 - 724
  • [3] Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR
    Rowe, Steven M.
    McColley, Susanna A.
    Rietschel, Ernst
    Li, Xiaolei
    Bell, Scott C.
    Konstan, Michael W.
    Marigowda, Gautham
    Waltz, David
    Boyle, Michael P.
    [J]. ANNALS OF THE AMERICAN THORACIC SOCIETY, 2017, 14 (02) : 213 - 219
  • [4] Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR
    Donaldson, Scott H.
    Pilewski, Joseph M.
    Griese, Matthias
    Cooke, Jon
    Viswanathan, Lakshmi
    Tullis, Elizabeth
    Davies, Jane C.
    Lekstrom-Himes, Julie A.
    Wang, Linda T.
    [J]. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2018, 197 (02) : 214 - 224
  • [5] An Open-Label Extension (OLE) study of Tezacaftor/Ivacaftor (TEZ/IVA) in patients (PTS) ≥ 12 years with cystic fibrosis (CF) Homozygous for F508DEL-CFTR (F/F) or Heterozygous for F508DEL-CFTR and a residual function mutation (F/RF)
    Flume, P.
    Biner, Fischer R.
    Downey, D. G.
    Brown, C.
    Jain, M.
    Fischer, R.
    de Boeck, C.
    Campbell, D.
    Wang, X.
    Ahluwalia, N.
    Owen, C. A.
    Wainwright, C.
    [J]. PNEUMOLOGIE, 2020, 74 : S87 - S87
  • [6] Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation
    De Stefano, Daniela
    Villella, Valeria R.
    Esposito, Speranza
    Tosco, Antonella
    Sepe, Angela
    De Gregorio, Fabiola
    Salvadori, Laura
    Grassia, Rosa
    Leone, Carlo A.
    De Rosa, Giuseppe
    Maiuri, Maria C.
    Pettoello-Mantovani, Massimo
    Guido, Stefano
    Bossi, Anna
    Zolin, Anna
    Venerando, Andrea
    Pinna, Lorenzo A.
    Mehta, Anil
    Bona, Gianni
    Kroemer, Guido
    Maiuri, Luigi
    Raia, Valeria
    [J]. AUTOPHAGY, 2014, 10 (11) : 2053 - 2074
  • [7] Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation
    Cholon, Deborah M.
    Esther, Charles R., Jr.
    Gentzsch, Martina
    [J]. EXPERT REVIEW OF PRECISION MEDICINE AND DRUG DEVELOPMENT, 2016, 1 (03): : 235 - 243
  • [8] Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR
    Taylor-Cousar, Jennifer L.
    Jain, Manu
    Barto, Tara Lynn
    Haddad, Tarik
    Atkinson, Jeffrey
    Tian, Simon
    Tang, Rui
    Marigowda, Gautham
    Waltz, David
    Pilewski, Joseph
    [J]. JOURNAL OF CYSTIC FIBROSIS, 2018, 17 (02) : 228 - 235
  • [9] Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR A Clinical Trial
    Sagel, Scott D.
    Khan, Umer
    Heltshe, Sonya L.
    Clancy, John P.
    Borowitz, Drucy
    Gelfond, Daniel
    Donaldson, Scott H.
    Moran, Antoinette
    Ratjen, Felix
    VanDalfsen, Jill M.
    Rowe, Steven M.
    [J]. ANNALS OF THE AMERICAN THORACIC SOCIETY, 2021, 18 (01) : 75 - 83
  • [10] CFTR MODULATION WITH TEZACAFTOR/IVACAFTOR IN PATIENTS HETEROZYGOUS FOR F508DEL AND A RESIDUAL FUNCTION MUTATION
    Rowe, Steven M.
    Davies, Jane
    [J]. PEDIATRIC PULMONOLOGY, 2017, 52 : S175 - S176
12345