A New Era for Cystic Fibrosis and Cystic Fibrosis Transmembrane Conductance Regulator Modulator Trials in Infants.
作者信息
Wainwright Claire E
机构信息
Paediatrics and Child Health University of Queensland South Brisbane, Queensland, Australia.
出版信息
Am J Respir Crit Care Med. 2022 Nov 15;206(10):1193-1195. doi: 10.1164/rccm.202207-1356ED.
Abstract
摘要
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本文引用的文献
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A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for .一项评估 Lumacaftor/Ivacaftor 在 1 岁以下囊性纤维化纯合子患儿中的疗效和安全性的 3 期、开放标签研究。
Am J Respir Crit Care Med. 2022 Nov 15;206(10):1239-1247. doi: 10.1164/rccm.202204-0734OC.
2
Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study.在囊性纤维化跨膜传导调节因子（CFTR）基因 F508del 纯合突变的 2-5 岁儿童中， lumacaftor-ivacaftor 的长期安全性：一项多中心、3 期、开放性、扩展研究。
Lancet Respir Med. 2021 Sep;9(9):977-988. doi: 10.1016/S2213-2600(21)00069-2. Epub 2021 May 6.
3
Determinants of lung disease progression measured by lung clearance index in children with cystic fibrosis.肺清除指数测定囊性纤维化患儿肺部疾病进展的决定因素。
Eur Respir J. 2021 Jul 8;58(1). doi: 10.1183/13993003.03380-2020. Print 2021 Jul.
4
CFTR-function and ventilation inhomogeneity in individuals with cystic fibrosis.囊性纤维化患者的CFTR功能与通气不均一性
J Cyst Fibros. 2021 Jul;20(4):641-647. doi: 10.1016/j.jcf.2020.12.009. Epub 2020 Dec 19.
5
CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine.CFTR调节剂：精准医学时代囊性纤维化的新面貌
Front Pharmacol. 2020 Feb 21;10:1662. doi: 10.3389/fphar.2019.01662. eCollection 2019.
6
Cystic fibrosis in the year 2020: A disease with a new face.2020年的囊性纤维化：面貌一新的疾病。
Acta Paediatr. 2020 May;109(5):893-899. doi: 10.1111/apa.15155. Epub 2020 Jan 22.
7
The future of cystic fibrosis care: a global perspective.囊性纤维化护理的未来：全球视角。
Lancet Respir Med. 2020 Jan;8(1):65-124. doi: 10.1016/S2213-2600(19)30337-6. Epub 2019 Sep 27.
8
Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor.来自美国和英国囊性纤维化注册中心的数据支持 CFTR 调节剂 ivacaftor 对疾病的修饰作用。
Thorax. 2018 Aug;73(8):731-740. doi: 10.1136/thoraxjnl-2017-210394. Epub 2018 May 10.
9
[Early lung disease in cystic fibrosis].[囊性纤维化中的早期肺部疾病]
Arch Pediatr. 2016 Dec;23(12S):12S9-12S14. doi: 10.1016/S0929-693X(17)30057-X.
10
Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study.在囊性纤维化和 CFTR 门控突变（KIWI）的 2-5 岁患者中，ivacaftor 的安全性、药代动力学和药效学：一项开放标签、单臂研究。
Lancet Respir Med. 2016 Feb;4(2):107-15. doi: 10.1016/S2213-2600(15)00545-7. Epub 2016 Jan 21.
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