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.