Family support for Tay-Sachs, Sandhoff, GM-1 and Canavan is provided by NTSAD and
we work closely with patient advocacy groups dedicated to various allied diseases.
Research efforts related to all lysosomal and
leukodystrophy allied diseases are supported by
NTSAD. The gained scientific knowledge is meaningful to all allied diseases because they share
many biochemical traits.
A genetic disease is a disorder
caused by an abnormality in DNA. There are many types of genetic
diseases. NTSAD focuses on inherited genetic diseases that fall into two
groups - lysosomal storage diseases and leukodystrophies.
Lysosomal Storage Diseases includes Tay-Sachs, Sandhoff and GM-1 diseases and are caused by a deficiency of an
enzyme in a
gene (Hex-A or GLB1).
Leukodystrophies are a group of rare genetic disorders, including Canavan disease, that affects the
central nervous system.
