SULPHATED SUGARS TO INCREASE ACTIVITY OF SULPHATASES EN LYSOSOMAL STORAGE DISORDERS

摘要

A method an preparation for increasing the activity of a sulphatase associated with a lysosomal storage disorder comprising administering a sulphated sugar. In particular useful for the lysosomal storage disorders associated with the following sulphatases:-iduronate-2-sulphatase, sulphamidase, glucosamine 6-sulphatase, galactose 6-sulphatase, galactosamine 4-sulphatase and galactose 3-sulphatase. The sulphated sugar is proposed to act as a pharmaceutical chaperone stabilizing the nascent sulphatase at the higher pH levels encountered in the endoplasmic reticulum (ER) and during transportation of the nascent sulphatase from the ER to the lysosome thereby avoiding the endoplasmic reticulum- associated degradation system. The sulphated sugar is preferably a monosaccharide thus not acting as a substrate and is released at the lower lysosomal pH levels where at least some activity is exhibited.