DERIVATIVES OF 1-DEOXI-GALACTONOJIRIMICIN AND ITS USE IN THE TREATMENT OF DISEASES RELATED TO LISOSOMAL STORAGE POTENTIATING ALFA-GALACTOSIDASE TO LISOSOMAL.

摘要

Use of an effective amount of a compound of the following formula: ** (See formula) ** in which R 1 represents H, CH 3, or CH 2 CH 3 R 2 and R 3 independently represent H, OH, an alkyl of 1-6 carbons , hydroxyalkyl, alkoxy, or a simple sugar; and R4 and R5 independently represent H or OH; or ** (See formula) ** in which for calistegina A 3: R 1 = H, R 2 = OH, R 3 = H, R 4 = H; for calistegine B2: R1 = H, R2 = OH, R3 = H, R4 = OH; for calistegine B 3: R 1 = H, R 2 = H, R 3 = OH, R 4 = OH; and for N-methyl-calistegine: R1 = CH3, R2 = OH, R3 = H, R4 = OH; in the manufacture of a medicament for treating a lysosomal glycosphingolipid storage disease, in which the compound is an enzyme inhibitor that is deficient in lysosomal glycosphingolipid storage disease.