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Tay-Sachs Disease: Treatments, Symptoms, Risks, and More - Healthline
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WebMar 3, 2024 · What is Tay-Sachs disease? Tay-Sachs is a rare disease of the central nervous system that most commonly affects infants. In infants and young children, it’s a progressive disease that is... WebWe’re thrilled to share the news that we have received U.S. FDA approval for AMVUTTRA (vutrisiran), the fifth #RNAi therapeutic developed by Alnyam… Liked by Monika Tasak Today is a major... WebMay 20, 2024 · Disease Overview Tay-Sachs disease is a rare, neurodegenerative disorder in which deficiency of an enzyme (hexosaminidase A) results in excessive accumulation of certain fats (lipids) known as gangliosides in the brain and nerve cells. This abnormal accumulation of gangliosides leads to progressive dysfunction of the central nervous … plugin emalware