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.
申请公布号
WO2005051331(A3)
申请公布日期
2006.03.02
申请号
WO2004US41345
申请日期
2004.11.24
申请人
ITY MOUNT SINAI SCHOOL OF MEDICINE OF NEW YORK UNIVERS;SCHUCHMAN, EDWARD, H.;DESNICK, ROBERT, J.