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Delivering Protein Therapeutics to the CNS for Gaucher Disease

Tay-Sachs is a genetic disorder caused by the absence of-hexosaminidase (HexA). This missing enzyme causes cells to become damaged, resulting in progressive neurological disorders. A definitive treatment for the symptoms of Tay-Sachs Disease does not yet exist.

There are 3 forms of Tay-Sachs:

The form is determined by the age of the individual when symptoms first appear. Only one form of Tay-Sachs occurs in a family. If a child has Infantile, older siblings are not at risk to develop Juvenile or Late Onset Tay-Sachs later in life.

Classic Infantile Tay-Sachs -Symptoms appear around 6 months of age

Juvenile Tay-Sachs -Symptoms typically appear between ages 2 and 5, but can occur anytime during childhood.

Late Onset Tay-Sachs -Symptoms typically appear in adolescence or early adulthood, but can appear later.