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WORLDSymposium 2022 | Feb 7, 2022 - Feb 11, 2022
GM2 gangliosidosis 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. β-hexosaminidase deficiency results in lysosomal accumulation of its substrate, GM2 ganglioside, in brain and nerve cells.
