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Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

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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. 

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