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 |
AU2010286550(B2) |
申请公布日期 |
2016.05.19 |
申请号 |
AU20100286550 |
申请日期 |
2010.08.28 |
申请人 |
MOUNT SINAI SCHOOL OF MEDICINE;GENZYME CORPORATION |
发明人 |
SCHUCHMAN, EDWARD H.;DESNICK, ROBERT J.;COX, GERALD F.;ANDREWS, LAURA P.;MURRAY, JAMES M. |
分类号 |
A61K38/43 |
主分类号 |
A61K38/43 |
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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