Getting Near to "Closing the Gap" in the Pediatric Age Group for the First Personalized Treatment of Cystic Fibrosis.
作者信息
van Koningsbruggen-Rietschel Silke
机构信息
CF-Center Cologne Faculty of Medicine, University of Cologne Cologne, Germany.
出版信息
Am J Respir Crit Care Med. 2021 Mar 1;203(5):536-537. doi: 10.1164/rccm.202010-3848ED.
Abstract
摘要
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本文引用的文献
1
Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial.依伐卡托特在 4 至<12 个月患有囊性纤维化且存在门控突变婴儿中的应用。两项 3 期临床试验的结果。
Am J Respir Crit Care Med. 2021 Mar 1;203(5):585-593. doi: 10.1164/rccm.202008-3177OC.
2
An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB).KLIMB 研究：在 2-5 岁有 CFTR 门控突变的 CF 患儿中开展 ivacaftor 的开放标签扩展研究，以启动治疗。
J Cyst Fibros. 2019 Nov;18(6):838-843. doi: 10.1016/j.jcf.2019.03.009. Epub 2019 Apr 30.
3
Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine.囊性纤维化的新兴治疗方法。从基因编辑到个性化医疗。
Front Pharmacol. 2019 Feb 27;10:121. doi: 10.3389/fphar.2019.00121. eCollection 2019.
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Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study.依伐卡托特治疗 12 至<24 月龄伴有 CFTR 门控突变（ARRIVAL）的囊性纤维化患儿：一项 3 期单臂研究。
Lancet Respir Med. 2018 Jul;6(7):545-553. doi: 10.1016/S2213-2600(18)30202-9. Epub 2018 Jun 7.
5
Cystic fibrosis transmembrane conductance regulator protein (CFTR) expression in the developing human brain: comparative immunohistochemical study between patients with normal and mutated CFTR.囊性纤维化跨膜电导调节蛋白（CFTR）在发育中人类大脑中的表达：正常 CFTR 和突变 CFTR 患者的比较免疫组织化学研究。
J Histochem Cytochem. 2014 Nov;62(11):791-801. doi: 10.1369/0022155414546190. Epub 2014 Jul 25.
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Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial.评估 G551D-CFTR 突变且肺功能正常的囊性纤维化患者使用 ivacaftor 的临床反应与肺清除指数：一项随机对照试验。
Lancet Respir Med. 2013 Oct;1(8):630-638. doi: 10.1016/S2213-2600(13)70182-6. Epub 2013 Sep 10.
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N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.
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Drug metabolizing enzyme activities versus genetic variances for drug of clinical pharmacogenomic relevance.药物代谢酶活性与药物临床药物基因组学相关的遗传变异。
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