医学
疾病
重症监护医学
溶酶体贮存障碍
基质还原疗法
孤儿药
儿科
生物信息学
病理
酶替代疗法
生物
作者
Weijing Kong,Cheng Lu,Yingxue Ding,Yan Meng
标识
DOI:10.1016/j.ejphar.2022.175023
摘要
Gaucher disease (GD), the most common lysosomal disorders, is a rare autosomal recessive hereditary disease that is caused by deficiency of glucosylceramidase. For now, there are five approved therapies for GD, which are used to treat thousands of patients with GD. Despite success of approved therapies, many unresolved issues attract academic institutions and industry to develop potential therapies to resolve them. This paper updated the latest information about approved therapies and potential curative therapies.
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