Sugar derivatives comprising sulfur-containing moieties and methods of making same and methods of using the same for the treatment of MPS IIIC

摘要

Described herein are modified sugar, iminosugar and azasugar compounds and methods of making same. One or more of these modified compounds contain sulfates, sulfites, sulfamates and/or sulfonamides. Also described are pharmaceutical compositions/formulations comprising these compounds, as well as methods using these modified sugar compounds for the treatment of MPS IIIC (also known as Sanfilippo Type C).

主权项

1. A compound having a structure represented by formula III: wherein: A1 is acetyl;
R1 is H, arylalkyl, aryl, or C1-4 alkyl;X and Y are each independently O or a lone pair of electrons; andeach Z is O; or a pharmaceutically acceptable salt, or solvate thereof.