Effects of Lumacaftor/Ivacaftor in a Pediatric Cohort Homozygous for F508del-CFTR

被引：1
|
作者：
Egan, Marie E. [1 ,2 ]
机构：
[1] Yale Sch Med, Dept Pediat, New Haven, CT 06520 USA
[2] Yale Sch Med, Dept Cellular & Mol Physiol, New Haven, CT 06520 USA
来源：
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE | 2017年 / 195卷 / 07期
关键词：
CYSTIC-FIBROSIS; PHE508DEL CFTR; IVACAFTOR; MUTATION; TRIAL;
D O I：
10.1164/rccm.201611-2290ED
中图分类号：
R4 [临床医学];
学科分类号：
1002 ; 100602 ;
摘要：
引用
收藏
页码：849 / 850
页数：2
相关论文
共 50 条
  • [31] Response to: 'Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?' by Jones and Barry
    Elborn, J. Stuart
    Ramsey, Bonnie
    Wainwright, Claire
    Boyle, Michael
    THORAX, 2016, 71 (02) : 185 - 186
  • [32] Characterization of Nasal Potential Difference in cftr Knockout and F508del-CFTR Mice
    Saussereau, Emilie Lyne
    Roussel, Delphine
    Diallo, Siradiou
    Debarbieux, Laurent
    Edelman, Aleksander
    Sermet-Gaudelus, Isabelle
    PLOS ONE, 2013, 8 (03):
  • [33] Virtual Drug Repositioning as a Tool to Identify Natural Small Molecules That Synergize with Lumacaftor in F508del-CFTR Binding and Rescuing
    Fossa, Paola
    Uggeri, Matteo
    Orro, Alessandro
    Urbinati, Chiara
    Rondina, Alessandro
    Milanesi, Maria
    Pedemonte, Nicoletta
    Pesce, Emanuela
    Padoan, Rita
    Ford, Robert C.
    Meng, Xin
    Rusnati, Marco
    D'Ursi, Pasqualina
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2022, 23 (20)
  • [34] Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease
    Hubert, Dominique
    Chiron, Raphael
    Camara, Boubou
    Grenet, Dominique
    Prevotat, Anne
    Bassinet, Laurence
    Dominique, Stephane
    Rault, Gilles
    Macey, Julie
    Honore, Isabelle
    Kanaan, Reem
    Leroy, Sylvie
    Dufeu, Nadine Desmazes
    Burgel, Pierre-Regis
    JOURNAL OF CYSTIC FIBROSIS, 2017, 16 (03) : 388 - 391
  • [35] F508del-CFTR Rescue: A Matter of Cell Stress Response
    Nieddu, Erika
    Pollarolo, Benedetta
    Merello, Luisa
    Schenone, Silvia
    Mazzei, Mauro
    CURRENT PHARMACEUTICAL DESIGN, 2013, 19 (19) : 3476 - 3496
  • [36] Targeting F508del-CFTR to develop rational new therapies for cystic fibrosis
    Cai, Zhi-wei
    Liu, Jia
    Li, Hong-yu
    Sheppard, David N.
    ACTA PHARMACOLOGICA SINICA, 2011, 32 (06) : 693 - 701
  • [37] EFFECT OF LUMACAFTOR IN COMBINATION WITH IVACAFTOR IN PATIENTS WITH CF WHO ARE HOMOZYGOUS FOR ΔF508-CFTR: PHASE 3 TRAFFIC AND TRANSPORT STUDIES
    Wainwright, Claire E.
    Elborn, J. Stuart
    Ramsey, Bonnie
    Huang, Xiaohong
    Marigowda, Gautham
    Waltz, David
    Boyle, Michael P.
    PEDIATRIC PULMONOLOGY, 2014, 49 : 156 - 157
  • [38] Corticosteroid use and increased CXCR2 levels on leukocytes are associated with lumacaftor/ivacaftor discontinuation in cystic fibrosis patients homozygous for the F508del CFTR mutation
    Pohl, Kerstin
    Nichols, David P.
    Taylor-Cousar, Jennifer L.
    Saavedra, Milene T.
    Strand, Matthew J.
    Nick, Jerry A.
    Bratcher, Preston E.
    PLOS ONE, 2018, 13 (12):
  • [39] S-Nitrosylation of CHIP Enhances F508Del-CFTR Maturation
    Zaman, Khalequz
    Knight, Julia
    Hussain, Faraaz
    Cao, Ruofan
    Estabrooks, Samuel K.
    Altawallbeh, Ghaith
    Holloway, Kristopher
    Jafri, Anjum
    Sawczak, Victoria
    Li, Yuejin
    Getsy, Paulina
    Sun, Fei
    Raffay, Thomas
    Cotton, Calvin
    Brodsky, Jeffrey L.
    Periasamy, Ammasi
    Lewis, Stephen J.
    Gaston, Benjamin
    AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY, 2019, 61 (06) : 765 - 775
  • [40] 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
    AUTOPHAGY, 2014, 10 (11) : 2053 - 2074
12345