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用于黏多糖贮积症治疗的酶替代疗法。

Enzyme replacement therapy for the management of the mucopolysaccharidoses.

作者信息

Wraith J E

机构信息

Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, Manchester, UK.

出版信息

Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S63-5. doi: 10.5414/cpp47063.

DOI:10.5414/cpp47063
PMID:20040314
Abstract

Enzyme replacement therapy (ERT) is now available for several of the mucopolysaccharidosis disorders. This brief review summarizes the role of ERT in reducing the burden of peripheral disease in many patients with mucopolysaccharidosis disorders, and describes the challenges that remain in treating the neurological manifestations of these conditions.

摘要

酶替代疗法（ERT）现已可用于多种黏多糖贮积症。本简要综述总结了ERT在减轻许多黏多糖贮积症患者外周疾病负担方面的作用，并描述了在治疗这些病症的神经学表现方面仍然存在的挑战。

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引用本文的文献

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A selective screening program for the early detection of mucopolysaccharidosis: Results of the FIND project - a 2-year follow-up study.一项用于黏多糖贮积症早期检测的选择性筛查项目：FIND项目的结果——一项为期2年的随访研究。
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