发明名称 |
CYCLODEXTRIN FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES |
摘要 |
The invention provides for methods of treating lysosomal storage disorders and/or reduction of non-cholesterol lipids, using cyclodextrin compounds, including in combination with other therapeutics, including vitamin E. |
申请公布号 |
US2015216895(A1) |
申请公布日期 |
2015.08.06 |
申请号 |
US201314419471 |
申请日期 |
2013.08.03 |
申请人 |
The United States of America, as represented by the Secretary, Department of Health & Human Servic |
发明人 |
McKew John;Zheng Wei;Xu Miao;Swaroop Manju;Marugan Juan J. |
分类号 |
A61K31/724;C08B37/16;G01N33/92;A61K31/355 |
主分类号 |
A61K31/724 |
代理机构 |
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代理人 |
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主权项 |
1. A method of treating a lysosomal storage disorder in a subject, comprising:
determining that the subject is in need of non-cholesterol lipid reduction or reduction of non-cholesterol dominant lipid and macromolecule accumulation, and administering to the subject in need thereof; an effective amount of a cyclodextrin compound, or a pharmaceutically acceptable salt, ester, solvate or hydrate thereof. |
地址 |
Rockville MD US |