Substrate Reduction Therapy

Substrate reduction therapy is an approach to treating certain metabolic disorders, especially glycogen storage diseases and lysosomal storage disorders such as Gaucher's disease, Tay-Sachs disease, Sandhoff disease and Sanfilippo syndrome. In these storage disorders, a failure in a metabolic pathway prevents the normal breakdown and disposal of a large molecule, and if the body's other pathways cannot compensate, the molecule builds up inside cells and eventually interferes with normal biological processes; substrate reduction therapy works by lowering the production of that molecule.