Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study.
作者信息
Hughes Derralynn A, Nicholls Kathleen, Sunder-Plassmann Gere, Jovanovic Ana, Feldt-Rasmussen Ulla, Schiffmann Raphael, Giugliani Robert, Jain Vipul, Viereck Chris, Castelli Jeffrey P, Skuban Nina, Barth Jay A, Bichet Daniel G
机构信息
Lysosomal Storage Disorders Unit, Royal Free NHS Foundation Trust and University College London, London, United Kingdom.
Department of Nephrology, Royal Melbourne Hospital, University of Melbourne, Parkville, Victoria, Australia.
出版信息
Am J Med Genet A. 2019 Jun;179(6):1069-1073. doi: 10.1002/ajmg.a.61105. Epub 2019 Mar 28.
Abstract
摘要
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本文引用的文献
1
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.法布里病中口服药理学伴侣米加司他与酶替代疗法的比较：III期随机ATTRACT研究的18个月结果
J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10.
2
Fabry disease: A pharmacological chaperone on the horizon.法布里病：即将出现的一种药物伴侣分子。
Nat Rev Nephrol. 2016 Nov;12(11):653-654. doi: 10.1038/nrneph.2016.138. Epub 2016 Sep 26.
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Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.米加司他治疗法布里病的疗效。
N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198.
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Fabry disease.法布里病。
Orphanet J Rare Dis. 2010 Nov 22;5:30. doi: 10.1186/1750-1172-5-30.
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Fabry disease: a review of current management strategies.法布瑞病：当前治疗策略综述。
QJM. 2010 Sep;103(9):641-59. doi: 10.1093/qjmed/hcq117. Epub 2010 Jul 21.
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Pharmacological chaperone therapy by active-site-specific chaperones in Fabry disease: in vitro and preclinical studies.法布里病中活性位点特异性伴侣分子的药理伴侣疗法：体外和临床前研究
Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S111-7.
7
The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.药物伴侣 1-脱氧半乳糖基神经酰胺可降低法布里病小鼠模型组织中的神经节苷脂 GM3 水平。
Mol Ther. 2010 Jan;18(1):23-33. doi: 10.1038/mt.2009.220. Epub 2009 Sep 22.
8
Life expectancy and cause of death in males and females with Fabry disease: findings from the Fabry Registry.法布瑞病患者的预期寿命和死亡原因：法布瑞登记研究结果。
Genet Med. 2009 Nov;11(11):790-6. doi: 10.1097/GIM.0b013e3181bb05bb.
9
The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines.药理学伴侣1-脱氧半乳糖野尻霉素可提高法布里病患者细胞系中α-半乳糖苷酶A的水平。
J Inherit Metab Dis. 2009 Jun;32(3):424-40. doi: 10.1007/s10545-009-1077-0. Epub 2009 Apr 18.
10
Mutant alpha-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin.在具有残余酶活性的法布里病患者中鉴定出的突变α-半乳糖苷酶A酶：生化特性及1-脱氧半乳糖野尻霉素对正常细胞内加工的恢复作用
Biochem J. 2007 Sep 1;406(2):285-95. doi: 10.1042/BJ20070479.
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