Reply to Martin and Grasemann: Is There a Role for Lumacaftor/Ivacaftor in Young Children with Cystic Fibrosis?
作者信息
Stahl Mirjam, Röhmel Jobst, Wielpütz Mark O, Mall Marcus A
机构信息
Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin Berlin, Germany.
German Center for Lung Research, associated partner site Berlin Berlin, Germany.
出版信息
Ann Am Thorac Soc. 2025 Mar;22(3):469-470. doi: 10.1513/AnnalsATS.202411-1237LE.
Abstract
摘要
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Reply to Martin and Grasemann: Is There a Role for Lumacaftor/Ivacaftor in Young Children with Cystic Fibrosis?回复马丁和格拉泽曼：鲁马卡托/依伐卡托对患有囊性纤维化的幼儿有作用吗？
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本文引用的文献
1
Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2-5 Years of Age Homozygous for : A Phase 2, Open-Label Clinical Trial.鲁马卡托/依伐卡托治疗对2至5岁纯合子囊性纤维化患儿疾病进展的长期影响：一项2期开放标签临床试验。
Ann Am Thorac Soc. 2024 Nov;21(11):1550-1559. doi: 10.1513/AnnalsATS.202402-201OC.
2
Effects of lumacaftor-ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2-11 years.鲁马卡托-依伐卡托疗法对2至11岁F508del纯合子囊性纤维化患者囊性纤维化跨膜传导调节因子功能的影响。
Front Pharmacol. 2023 May 30;14:1188051. doi: 10.3389/fphar.2023.1188051. eCollection 2023.
3
Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for : A Phase 2 Placebo-controlled Clinical Trial.Lumacaftor/Ivacaftor 对 2 至 5 岁纯合子囊性纤维化患儿疾病进展的影响：一项 2 期安慰剂对照临床试验。
Ann Am Thorac Soc. 2023 Aug;20(8):1144-1155. doi: 10.1513/AnnalsATS.202208-684OC.
4
Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two Alleles.依伐卡托/泰它卡托/艾氟康唑治疗对具有一个或两个等位基因的囊性纤维化患者 CFTR 功能的影响。
Am J Respir Crit Care Med. 2022 Mar 1;205(5):540-549. doi: 10.1164/rccm.202110-2249OC.
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Effects of Lumacaftor-Ivacaftor Therapy on Cystic Fibrosis Transmembrane Conductance Regulator Function in Phe508del Homozygous Patients with Cystic Fibrosis.利那洛肽治疗在囊性纤维化跨膜电导调节因子功能对囊性纤维化纯合子 Phe508del 患者的影响。
Am J Respir Crit Care Med. 2018 Jun 1;197(11):1433-1442. doi: 10.1164/rccm.201710-1983OC.
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Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.针对携带Phe508del CFTR纯合突变的囊性纤维化患者使用鲁马卡托-依伐卡托。
N Engl J Med. 2015 Oct 29;373(18):1783-4. doi: 10.1056/NEJMc1510466.
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