Invention Grant
- Patent Title: Chaperone-based therapy for Niemann-Pick disease
- Patent Title (中): 基于伴侣疗法的尼曼 - 皮克病
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Application No.: US10998270Application Date: 2004-11-24
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Publication No.: US07750050B2Publication Date: 2010-07-06
- Inventor: Edward H. Schuchman , Robert J. Desnick
- Applicant: Edward H. Schuchman , Robert J. Desnick
- Applicant Address: US NY New York
- Assignee: Mount Sinai School of Medicine of New York University
- Current Assignee: Mount Sinai School of Medicine of New York University
- Current Assignee Address: US NY New York
- Agency: Fish & RIchardson P.C.
- Main IPC: A61K31/16
- IPC: A61K31/16 ; C12N9/99
Abstract:
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.
Public/Granted literature
- US20050153934A1 Chaperone-based therapy for Niemann-Pick disease Public/Granted day:2005-07-14
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