Real-world data assessment of safety of home-based and hospital/outpatient-based laronidase enzyme replacement therapy for mucopolysaccharidosis I.
作者信息
Wilson Amanda, Colzani Raffaella M, Flores Ana Lorena
机构信息
Sanofi Genzyme, USA.
出版信息
Mol Genet Metab Rep. 2021 Mar 4;27:100736. doi: 10.1016/j.ymgmr.2021.100736. eCollection 2021 Jun.
Abstract
摘要
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Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis.静脉注射拉罗尼酶治疗黏多糖贮积症 I 型的疗效和安全性：一项系统评价和荟萃分析。
PLoS One. 2017 Aug 31;12(8):e0184065. doi: 10.1371/journal.pone.0184065. eCollection 2017.
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Home infusion program with enzyme replacement therapy for Fabry disease: The experience of a large Italian collaborative group.法布里病酶替代疗法的家庭输注方案：意大利大型协作组的经验
Mol Genet Metab Rep. 2017 Jun 22;12:85-91. doi: 10.1016/j.ymgmr.2017.06.005. eCollection 2017 Sep.
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The natural history of MPS I: global perspectives from the MPS I Registry.黏多糖贮积症 I 型的自然病史：来自黏多糖贮积症 I 型注册库的全球视角
Genet Med. 2014 Oct;16(10):759-65. doi: 10.1038/gim.2014.25. Epub 2014 Mar 27.
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Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey.采用伊杜硫酸酯酶静脉酶替代疗法进行家庭治疗黏多糖贮积症 II 型 - 亨特结果调查数据。
Mol Genet Metab. 2010 Oct-Nov;101(2-3):123-9. doi: 10.1016/j.ymgme.2010.06.011. Epub 2010 Jun 23.
5
Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe.采用酶替代疗法对I型黏多糖贮积症进行居家治疗是可行且安全的。
J Inherit Metab Dis. 2007 Nov;30(6):984. doi: 10.1007/s10545-007-0686-8. Epub 2007 Sep 21.
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