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专利概况
专利名称 Methods for increasing intracellular activity of Hsp70
申请号 US15600407 申请日
公开(公告)号 US10532085B2 公开(公告)日
申请(专利权)人 Orphazyme A/S 发明人 Thomas K Jensen; Anders M鴕keberg Hinsby
专利来源 国家知识产权局 转化方式
摘要

The present invention relates to a bioactive agent capable of increasing the intracellular concentration and/or activity of Hsp70 for use in the treatment of a lysosomal storage disease which arise from a defect in an enzyme whose activity is not directly associated with the presence of lysosomal BMP as a co-factor; such as glycogen storage diseases, gangliosidoses, neuronal ceroid lipofuscinoses, cerebrotendinous cholesterosis, Wolman' s disease, cholesteryl ester storage disease, disorders of glycosaminoglycan metabolism, mucopolysaccharidoses, disorders of glycoprotein metabolism, mucolipidoses, aspartylglucosaminuria, fucosidosis, mannosidoses, and sialidosis type II.

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