Sandhoff disease is very similar to Tay-Sachs disease, although it occurs in slightly different groups of people in the population. The condition has absolutely similar effects, but it is less well known because of the condition being only recognized in the 1960s as a variant of Tay-Sachs disease.

The effects in infants and children are practically the same and its dreadful effects are indistinguishable at the bedside. Mainly it is in Sandhoff disease models that gene therapy and other enhancing treatments have been first tried; they respond well to gene therapy, which is also being extended to Tay-Sachs disease as well.

A similar number of patients are affected today, and they come from all over the world; they and their families would be enormously grateful for any token of support for the provision of research funds to help advance treatment for this condition, which is running in parallel with the gene therapy programme for Tay-Sachs disease.