NTSAD provides family services, research, and education for
Tay-Sachs, GM-1, Sandhoff, Canavan and allied diseases.
Join our cause to wipe out these genetic diseases.
Enzyme Replacement Therapy (ERT) provides the missing or deficient enzyme through regular IV infusions. However, enzymes are large molecules that do not pass the blood brain barrier into the central nervous system. Approved ERT therapies are currently only effective for treating the non-neurological symptoms of diseases.