发明名称 |
A method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives |
摘要 |
The present invention provides a method for increasing the activity of a mutant or wild-type α-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1- deoxynojirimycin. The invention also provides a method for the treatment of Pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. The 1-deoxynojirimycin derivative is substituted at the N or Cl position. Combination therapy with replacement α-glucosidase gene or enzyme is also provided. |
申请公布号 |
AU2006247065(B2) |
申请公布日期 |
2012.03.22 |
申请号 |
AU20060247065 |
申请日期 |
2006.05.17 |
申请人 |
AMICUS THERAPEUTICS INC. |
发明人 |
BOYD, ROBERT;LEE, GARY;SHETH, KAMLESH;ZHU, XIAOXIANG;MUGRAGE, BENJAMIN |
分类号 |
A61K48/00 |
主分类号 |
A61K48/00 |
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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