Is a
Patent attributes
Patent Jurisdiction
Patent Number
Date of Patent
July 6, 2010
Patent Application Number
10998270
Date Filed
November 24, 2004
Patent Citations Received
Patent Primary Examiner
Patent 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.
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