发明授权
- 专利标题： Chaperone-based therapy for Niemann-Pick disease
- 专利标题（中）： 基于伴侣疗法的尼曼 - 皮克病
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申请号： US10998270申请日： 2004-11-24
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公开(公告)号： US07750050B2公开(公告)日： 2010-07-06
- 发明人: Edward H. Schuchman , Robert J. Desnick
- 申请人： Edward H. Schuchman , Robert J. Desnick
- 申请人地址： US NY New York
- 专利权人： Mount Sinai School of Medicine of New York University
- 当前专利权人： Mount Sinai School of Medicine of New York University
- 当前专利权人地址： US NY New York
- 代理机构： Fish & RIchardson P.C.
- 主分类号： A61K31/16
- IPC分类号： A61K31/16 ; C12N9/99
摘要：
The present invention provides a method for treating individuals affected with the acid sphingomyelinase-deficient forms of Niemann-Pick disease (i.e., Type A or Type B Niemann-Pick) by administering small molecules as specific molecular “chaperones” for the deficient acid sphingomyelinase (ASM) enzyme associated with the disease. The molecules are ceramide, sphingomyelin, or phosphonucleotide analogues.
公开/授权文献
- US20050153934A1 Chaperone-based therapy for Niemann-Pick disease 公开/授权日：2005-07-14
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