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Velaglucerase alfa.

作者信息

Aerts Johannes M F G, Yasothan Uma, Kirkpatrick Peter

出版信息

Nat Rev Drug Discov. 2010 Nov;9(11):837-8. doi: 10.1038/nrd3311. Epub 2010 Oct 29.

DOI:10.1038/nrd3311
PMID:21030995
Abstract
摘要

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Velaglucerase alfa.维拉苷酶α
Nat Rev Drug Discov. 2010 Nov;9(11):837-8. doi: 10.1038/nrd3311. Epub 2010 Oct 29.
2
Velaglucerase alfa for the management of type 1 Gaucher disease.依洛硫酸酯酶α治疗戈谢病 1 型。
Clin Ther. 2012 Feb;34(2):259-71. doi: 10.1016/j.clinthera.2011.12.017. Epub 2012 Jan 20.
3
Velaglucerase alfa: a new option for Gaucher disease treatment.维拉苷酶α：戈谢病治疗的新选择。
Drugs Today (Barc). 2011 Jul;47(7):515-29. doi: 10.1358/dot.2011.47.7.1608922.
4
Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease.开发一组高灵敏度、等效的检测试剂盒，用于检测戈谢病患者对 velaglucerase alfa 或 imiglucerase 酶替代疗法的抗体反应。
J Immunol Methods. 2011 Oct 28;373(1-2):45-53. doi: 10.1016/j.jim.2011.07.020. Epub 2011 Aug 6.
5
Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease.维拉苷酶阿尔法酶替代疗法与伊米苷酶治疗戈谢病的比较。
Am J Hematol. 2013 Mar;88(3):179-84. doi: 10.1002/ajh.23382. Epub 2013 Feb 9.
6
Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.长期使用维拉苷酶α治疗1型戈谢病初治或曾接受伊米苷酶治疗的儿童。
Mol Genet Metab. 2016 Feb;117(2):164-71. doi: 10.1016/j.ymgme.2015.05.012. Epub 2015 Jun 1.
7
A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability.一项多中心、开放性标签的维拉苷酶α酶替代治疗戈谢病 1 型患者的治疗方案（HGT-GCB-058）：安全性和耐受性。
Genet Med. 2014 May;16(5):359-66. doi: 10.1038/gim.2013.154. Epub 2013 Nov 21.
8
Effects of switching from imiglucerase to velaglucerase alfa without dose reduction nor wash out in type 1 Gaucher disease.在1型戈谢病患者中，不降低剂量且不进行洗脱期，从伊米苷酶转换为维拉苷酶α的效果。
Blood Cells Mol Dis. 2014 Jun-Aug;53(1-2):94-6. doi: 10.1016/j.bcmd.2013.12.004. Epub 2014 Jan 7.
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Development of anti-velaglucerase alfa antibodies in clinical trial-treated patients with Gaucher disease.在接受临床试验治疗的戈谢病患者中抗维拉苷酶α抗体的产生
Blood Cells Mol Dis. 2016 Jul;59:37-43. doi: 10.1016/j.bcmd.2016.03.004. Epub 2016 Mar 5.
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A comparison study of bioanalytical methods for detection and characterization of anti-velaglucerase alfa antibodies.
Bioanalysis. 2017 May;9(10):775-786. doi: 10.4155/bio-2016-0274. Epub 2017 Apr 28.

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Investigations on therapeutic glucocerebrosidases through paired detection with fluorescent activity-based probes.

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1
Early achievement and maintenance of the therapeutic goals using velaglucerase alfa in type 1 Gaucher disease.使用威拉葡萄糖脑苷脂酶 α 在 1 型 Gaucher 病中实现早期治疗目标并维持疗效。
Blood Cells Mol Dis. 2011 Jan 15;46(1):119-23. doi: 10.1016/j.bcmd.2010.07.008. Epub 2010 Aug 19.
2
Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study.口服伊米苷酶（Genz-112638）治疗戈谢病 1 型：2 年 2 期研究的血液学、内脏和骨骼表现改善结果。
Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16.
3
通过基于荧光活性的探针进行配对检测对治疗性葡萄糖脑苷脂酶的研究。
PLoS One. 2017 Feb 16;12(2):e0170268. doi: 10.1371/journal.pone.0170268. eCollection 2017.
4
Direct site-specific glycoform identification and quantitative comparison of glycoprotein therapeutics: imiglucerase and velaglucerase alfa.糖蛋白疗法的直接位点特异性糖型鉴定及定量比较：伊米苷酶和维拉苷酶α
AAPS J. 2015 Mar;17(2):405-15. doi: 10.1208/s12248-014-9706-4. Epub 2014 Dec 13.
5
Endosperm-specific expression of human acid beta-glucosidase in a waxy rice.在糯稻中胚乳特异性表达人酸性β-葡萄糖苷酶。
Rice (N Y). 2012 Dec 6;5(1):34. doi: 10.1186/1939-8433-5-34.
6
Generation of a Chinese hamster ovary cell line producing recombinant human glucocerebrosidase.产生重组人葡萄糖脑苷脂酶的中国仓鼠卵巢细胞系的构建
J Biomed Biotechnol. 2012;2012:875383. doi: 10.1155/2012/875383. Epub 2012 Oct 3.
7
Gaucher disease and the synucleinopathies: refining the relationship.戈谢病与突触核蛋白病：关系的再审视。
Orphanet J Rare Dis. 2012 Jan 31;7:12. doi: 10.1186/1750-1172-7-12.
8
Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response.戈谢氏病患者血浆葡萄糖基神经酰胺升高与其表型、贮积细胞标志物和治疗反应的关系。
Blood. 2011 Oct 20;118(16):e118-27. doi: 10.1182/blood-2011-05-352971. Epub 2011 Aug 25.
9
Gaucher disease and cancer: concept and controversy.戈谢病与癌症：概念与争议
Int J Cell Biol. 2011;2011:150450. doi: 10.1155/2011/150450. Epub 2011 Jun 7.
10
Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.溶酶体贮积症诊断中的生物标志物：蛋白质、脂质和抑制物。
J Inherit Metab Dis. 2011 Jun;34(3):605-19. doi: 10.1007/s10545-011-9308-6. Epub 2011 Mar 29.
Short-term withdrawal from imiglucerase: what can we learn from it?
Blood Cells Mol Dis. 2011 Jan 15;46(1):105-6. doi: 10.1016/j.bcmd.2010.05.003. Epub 2010 Jun 1.
4
Enzyme replacement therapy "drug holiday": results from an unexpected shortage of an orphan drug supply in Australia.酶替代疗法“药物假期”：澳大利亚一种罕见病药物供应意外短缺的结果。
Blood Cells Mol Dis. 2011 Jan 15;46(1):107-10. doi: 10.1016/j.bcmd.2010.05.002. Epub 2010 Jun 1.
5
Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply.由于供应短缺而停止使用伊米苷酶治疗戈谢病时无骤变。
Blood Cells Mol Dis. 2011 Jan 15;46(1):111-4. doi: 10.1016/j.bcmd.2010.05.001. Epub 2010 Jun 9.
6
Comparative therapeutic effects of velaglucerase alfa and imiglucerase in a Gaucher disease mouse model.在 Gaucher 病小鼠模型中比较 velaglucerase alfa 和 imiglucerase 的治疗效果。
PLoS One. 2010 May 20;5(5):e10750. doi: 10.1371/journal.pone.0010750.
7
A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.一项关于伊曲康唑（Genz-112638）的 2 期研究，这是一种用于治疗 1 型戈谢病的口服底物减少疗法。
Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3.
8
Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience.1 型 Gaucher 病成人患者用 velaglucerase alfa 替代治疗的 1/2 期和扩展研究：48 个月经验。
Blood. 2010 Jun 10;115(23):4651-6. doi: 10.1182/blood-2010-02-268649. Epub 2010 Mar 18.
9
Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease.不可抗力：应对戈谢病患者伊米苷酶（思而赞）供应受限的治疗措施。
Blood Cells Mol Dis. 2010 Jan 15;44(1):41-7. doi: 10.1016/j.bcmd.2009.09.006. Epub 2009 Oct 4.
10
Characterization of gene-activated human acid-beta-glucosidase: crystal structure, glycan composition, and internalization into macrophages.基因激活的人酸性β-葡萄糖苷酶的表征：晶体结构、聚糖组成和巨噬细胞内吞作用。
Glycobiology. 2010 Jan;20(1):24-32. doi: 10.1093/glycob/cwp138. Epub 2009 Sep 9.