发明名称 |
ACID ALPHA-GLUCOSIDASE AND A β-2 AGONIST FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDER |
摘要 |
The present disclosure is directed to methods of treating a lysosomal storage disorder by administering a therapeutic agent as an adjunctive therapy to lysosomal enzyme replacement therapy. Other embodiments are directed to methods of increasing expression of a receptor for a lysosomal enzyme and methods of treating a lysosomal storage disorder. Some embodiments are directed to a method of treating a glycogen storage disease II in an individual by administering to the individual an acid alpha-glucosidase and a β2 agonist. Some embodiments herein are directed to a method of treating a glycogen storage disease II by administering (1) rhGAA; and (2) clenbuterol. |
申请公布号 |
WO2017049161(A1) |
申请公布日期 |
2017.03.23 |
申请号 |
WO2016US52254 |
申请日期 |
2016.09.16 |
申请人 |
DUKE UNIVERSITY |
发明人 |
KOEBERL, Dwight, D.;KISHNANI, Priya |
分类号 |
A61K31/7088;A61K31/711;A61K38/47 |
主分类号 |
A61K31/7088 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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