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CHAPERONE-BASED THERAPY FOR NIEMANN-PICK DISEASE
CHAPERONE-BASED THERAPY FOR NIEMANN-PICK DISEASE
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机译:基于伴侣的尼曼病的治疗
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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.
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