| 发明名称 |
COMPOUNDS FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSES |
| 摘要 |
<p>A method of treating mucopolysaccharidoses, which are part of the lysosomal storage disease family, comprising administering a compound that decreases activity of N-deacetylase/N-sulfotransferase (NDST) to a subject in need thereof. High throughput screens for NDSTl transcription inhibitors yield compounds including SAHA, givinostat and pyrimethamine.</p> |
| 申请公布号 |
WO2014172776(A1) |
申请公布日期 |
2014.10.30 |
| 申请号 |
WO2014CA00363 |
申请日期 |
2014.04.22 |
| 申请人 |
THE HOSPITAL FOR SICK CHILDREN;THE UNIVERSITY OF BRITISH COLUMBIA |
发明人 |
MAHURAN, DON;TKACHYOVA, ILONA;FAN, XIAOLIAN;CIUFOLINI, MARCO |
| 分类号 |
A61K31/505;A61K31/167;A61K31/27;A61K31/352;A61P3/00 |
主分类号 |
A61K31/505 |
| 代理机构 |
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