Methods for Treating Pompe Disease

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专利权人:

Jonathan LeBowitz;John Maga

发明人:

Jonathan LeBowitz,John Maga

申请号：

US13268369

公开号：

US20120093794A1

申请日:

2011.10.07

申请国别(地区):

US

年份:

2012

代理人:

摘要：

The present invention provides methods for treating Pompe disease in a subject by administering to the subject a therapeutically effective amount of a fusion protein which includes human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain. The lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.

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中国工程科技知识中心

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http://www.ckcest.cn/home/