FIELD: medicine; therapy.
SUBSTANCE: invention relates to the use of acetyleucine or a pharmaceutically acceptable salt thereof for the treatment of lysosomal storage disease (LSD) or one or more symptoms associated with LSN in a subject in need thereof, while LSD is not Niemann-Pick Type C disease. Preferably LSD is selected from glycogen storage disease, mucopolysaccharidosis, mucolipidosis, oligosaccharidosis, lipidosis, sphingolipidosis, lysosomal transfer diseases, lack of hydrolase of the primary lysosome, defect in post-translational processing of lysosomal enzymes, defect in the transfer of lysosomal enzyme proteins, lysosomal enzyme protection defect, lack of transmembrane (non-enzymatic) protein, unclassified defect, Tay-Sachs disease, AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick Type A disease, Niemann-Pick Type B disease, Fabry disease, neuronal ceroid lipofuscinosis, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulfatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria.
EFFECT: invention provides effective treatment of the specified diseases.
16 cl, 10 dwg, 15 tbl, 7 ex
