发明名称 |
DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY |
摘要 |
The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B. |
申请公布号 |
KR20120081077(A) |
申请公布日期 |
2012.07.18 |
申请号 |
KR20127005293 |
申请日期 |
2010.08.28 |
申请人 |
GENZYME CORPORATION;MOUNT SINAI SCHOOL OF MEDICINE |
发明人 |
SCHUCHMAN EDWARD H.;DESNICK ROBERT J.;COX GERALD F.;ANDREWS LAURA P.;MURRAY JAMES M. |
分类号 |
A61K38/46;A61P3/00;A61P3/06 |
主分类号 |
A61K38/46 |
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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