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 代理人
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