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compound and method of treating a lysosomal storage disease
专利权人:
ORPHAZYME APS
发明人:
ANDERS MORKEBERG HINSBY,THOMAS KIRKEGAARD JENSEN
申请号:
BR112013013143
公开号:
BR112013013143A2
申请日:
2011.11.22
申请国别(地区):
BR
年份:
2016
代理人:
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.
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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