| abstract |
A method is disclosed for the in vivo treatment of patients having a Lysosomal storage disease with a significant central nervous system (CNS) involvement. Said method comprises administration to said patient a small but storage-inhibitory effective amount of an N-alkyl derivative of 1,5-dideoxy-1,5-imino-D-galactitol, or an O-acylated pro-drug thereof, in which said alkyl group contains from about 2 to about 8 carbon atoms. The Lysosomal storage diseases having a significant CNS involvement are illustrated by Tay-Sachs disease, Sandhoff disease, GM1 gangliosidosis and Fabray disease. |