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
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