Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

The severity of a Lysosomal storage disorder depends on the amount of enzyme activity that is present, and where in the body the substrate (debris) is accumulating. If some enzyme activity is present, symptoms usually begin later on (as in juvenile or adult onset version of the diseases.) The most severe disorders are where the debris builds up in the central nervous system, such as in Tay Sachs, Sandhoff and GM-1.

 

Login