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USE OF ARIMOCLOMOL IN ACTIVATING CLEAR GENE EXPRESSION AS TREATMENT FOR LYSOSOMAL STORAGE DISORDERS
发明专利审中
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29权利要求 · 8 独立
§ Ⅰ
卷宗概要
发明人
Sven Guenther; Travis Mickle; Thomas Kirkegaard Jensen
IPC 分类
A61K 31/4545A61P 3/
CPC 分类
A61K31/4545A61P3/
The present disclosure provides methods of treating lysosomal storage disorders such as Niemann-Pick Disease, Type-C, by administering a therapeutically effective amount of arimoclomol. The present disclosure further provides methods of removing lysosomal cholesterol by administering the same.
原文（中文）
The present disclosure provides methods of treating lysosomal storage disorders such as Niemann-Pick Disease, Type-C, by administering a therapeutically effective amount of arimoclomol. The present disclosure further provides methods of removing lysosomal cholesterol by administering the same.