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High concentration α-glucosidase compositions for the treatment of Pompe disease
专利权人:
Inc.;Amicus Therapeutics
发明人:
Kenneth Valenzano,John Crowley,Richie Khanna,John Flanagan
申请号:
US14106670
公开号:
US09303249B2
申请日:
2013.12.13
申请国别(地区):
US
年份:
2016
代理人:
摘要:
The present application provides for compositions comprising high concentrations of acid α-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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