Invention Grant
- Patent Title: Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
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Application No.: US12870790Application Date: 2010-08-28
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Publication No.: US08349319B2Publication Date: 2013-01-08
- Inventor: Edward H. Schuchman , Robert J. Desnick , Gerald F. Cox , Laura P. Andrews , James M. Murray
- Applicant: Edward H. Schuchman , Robert J. Desnick , Gerald F. Cox , Laura P. Andrews , James M. Murray
- Applicant Address: US NY New York US MA Cambridge
- Assignee: Mount Sinai School of Medicine,Genzyme Corporation
- Current Assignee: Mount Sinai School of Medicine,Genzyme Corporation
- Current Assignee Address: US NY New York US MA Cambridge
- Agency: Jones Day
- Main IPC: A61K38/46
- IPC: A61K38/46

Abstract:
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.
Public/Granted literature
- US20110052559A1 DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY Public/Granted day:2011-03-03
Information query
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