Tay-Sachs, Sandhoff and GM1 are rare genetic disorders (also known as lysosomal storage disorders) that affect the central nervous system. Infants and children born with these diseases appear healthy at birth but progressively lose all motor function and typically die between five and 10 years old. The Late Onset forms of Tay-Sachs and Sandhoff diseases are progressively debilitating but not typically fatal. Canavan disease is a rare genetic disease (also known as a leukodystrophy) that causes the progressive deterioration of all motor function.
There is no cure or treatment for any of these rare genetic diseases. However, research is closer, more than ever, to developing treatments and even potential cures. In order to speed the way for treatments, funds must be raised to subsidize the outstanding work being conducted worldwide. After nearly 60 years NTSAD remains one of the oldest patient advocacy organizations committed to caring for families and finding a cure.
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