Response to Letter to the Editor on "Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options".
作者信息
Van Rossum Alison, Holsopple Megan
机构信息
University of Florida Health Jacksonville, FL, USA.
Froedtert & the Medical College of Wisconsin, Milwaukee, WI, USA.
出版信息
Hosp Pharm. 2017 Dec;52(11):727-728. doi: 10.1177/0018578717737135. Epub 2017 Nov 9.
Abstract
摘要
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本文引用的文献
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Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.在接受酶替代疗法病情已稳定的1型戈谢病患者中， eliglustat可维持长期临床稳定性。
Blood. 2017 Apr 27;129(17):2375-2383. doi: 10.1182/blood-2016-12-758409. Epub 2017 Feb 6.
2
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.
3
Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.口服依利格鲁司他对1型戈谢病患者脾肿大的影响：ENGAGE随机临床试验
JAMA. 2015 Feb 17;313(7):695-706. doi: 10.1001/jama.2015.459.
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Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher disease.接受维拉苷酶α治疗戈谢病的女性患者的妊娠结局。
J Obstet Gynaecol Res. 2014 Apr;40(4):968-75. doi: 10.1111/jog.12254. Epub 2014 Feb 26.
5
Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.植物细胞表达重组葡萄糖脑苷脂酶（taliglucerase alfa）的关键性试验：一种新型戈谢病酶替代治疗药物。
Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6.
6
Changes in the atherogenic profile of patients with type 1 Gaucher disease after miglustat therapy.戈谢病 1 型患者米格列醇治疗后动脉粥样硬化形成相关指标的变化。
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