Treatment options for patients with Gaucher disease

Rabah M. Shawky; Solaf M. Elsayed

首页> 外文期刊>Egyptian Journal of Medical Human Genetics >Treatment options for patients with Gaucher disease

【24h】

Treatment options for patients with Gaucher disease

机译：高雪氏病患者的治疗选择

获取原文

掌桥外文数据库（机构版） >>

开具论文收录证明 >>

文献代查 >>

页面导航

摘要
著录项
相似文献
相关主题

摘要

Gaucher disease is the most common lysosomal storage disorder due to deficiency of ?-glucocerebrosidase. Since the introduction of Ceredase in 1991, enzyme replacement therapy has been the mainstay of treatment with its major disadvantage of long life dependency on biweekly IV therapy. It was more than a decade later when the substrate reduction therapy – an oral treatment – was approved for Gaucher disease. Future therapeutic modalities will include pharmacological chaperon and possibly gene therapy. The aim of this review is to high light the current and future treatment options for patients with Gaucher disease and to compare their effects and side effects.

机译：由于缺乏β-葡萄糖脑苷脂酶，戈谢病是最常见的溶酶体贮积病。自1991年引入Ceredase以来，酶替代疗法一直是治疗的主要手段，其主要缺点是依赖于双周一次的IV疗法寿命长。十多年后，减少基质减少疗法（一种口服疗法）被批准用于高雪氏病。未来的治疗方式将包括药理伴侣和可能的基因治疗。这篇综述的目的是突出针对高雪氏病患者的当前和未来治疗方案，并比较其效果和副作用。

著录项

来源
《Egyptian Journal of Medical Human Genetics》 |2016年第3期|共5页
作者
Rabah M. Shawky; Solaf M. Elsayed;
展开▼
作者单位

展开▼
收录信息
原文格式 PDF
正文语种
中图分类遗传学;
关键词

相似文献

外文文献
中文文献
专利

1. Clinical response to eliglustat in treatment-na?ve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry [J] . Jennifer Ibrahim, Lisa H. Underhill, John S. Taylor, Molecular Genetics and Metabolism Reports . 2016,第1期

机译：国际合作Gaucher Group Gaucher Registry登记的初治Gaucher疾病类型1的初治患者对eliglustat的临床反应：事后比较与伊米苷酶治疗的患者
2. Exploring the patient journey to diagnosis of Gaucher disease from the perspective of 212 patients with Gaucher disease and 16 Gaucher expert physicians [J] . Atul Mehta, Nadia Belmatoug, Bruno Bembi, Molecular genetics and metabolism . 2017,第3期

机译：从212例GAUCHER病和16号GAUCHER专家医师探索患者诊断GAUCHER病的诊断
3. Treatment patterns from 647 patients with Gaucher disease: An analysis from the Gaucher Outcome Survey [J] . Deegan P., Fernandez-Sasso D., Giraldo P., Blood cells, molecules and diseases . 2018,第期

机译：来自647例Gaucher病患者的治疗模式：Gaucher结果调查分析
4. FT-ICR MS Profiling of Small Molecule Derived from Plasma Obtained from Gaucher Disease Patients [C] . TuKiet T. Lam, Mei Yang, Ji Young Lee, American Society for Mass Spectrometry Conference on Mass Spectrometry and Allied Topics . 2009

机译：FT-ICR MS谱分析来自Gaucher病患者获得的血浆衍生的小分子
5. Patients' opinions on genetic counseling on the increased risk of Parkinson's disease among Gaucher disease carriers [D] . Mulhern, Maureen 2015

机译：患者对高彻病携带者中帕金森氏病风险增加的遗传咨询意见
6. Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry [O] . Jennifer Ibrahim, Lisa H. Underhill, John S. Taylor, 2016

机译：国际合作Gaucher集团Gaucher注册中心的未接受过Gaucher疾病类型1的初治患者对依格鲁司特的临床反应：事后比较与伊米苷酶治疗的患者
7. Treatment options for patients with Gaucher disease [O] . Shawky Rabah M., Elsayed Solaf M. 2016

机译：高雪氏病患者的治疗选择

Treatment options for patients with Gaucher disease

摘要

著录项

相似文献

相关主题

期刊订阅