Press Releases from Tay-Sachs Disease Therapeutics- Pharma Proff (1 total)
Tay-Sachs Disease Therapeutics- Pipeline Analysis 2018, Clinical Trials & Result …
Tay-Sachs is a lysosomal storage disorder caused by the deficiency of vital enzymes called beta-hexosaminidase A (Hex-A). The role of Hex-A is to degrade a fatty substance or lipid called GM-2 ganglioside. In the absence of the Hex-A enzymes, GM-2 accumulates abnormally in cells, especially in the nerve cells or neurons, of the brain.
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In children, the destructive process begins in the fetus early in pregnancy.…
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