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WORLDSymposium 2025 | Feb 3, 2025 - Feb 7, 2025
GM2 gangliosidoses comprises rare, autosomal recessive, neurodegenerative lysosomal storage disorders, primarily Tay-Sachs disease and Sandhoff disease. Pathogenic variants lead to a deficiency in the activity of the enzyme (β-hexosaminidase) responsible for catabolizing GM2 ganglioside. This causes lysosomal accumulation of its substrate in brain and nerve cells.
