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Sandhoff disease
A lysosomal disorder from the GM2 gangliosidosis family, caused by biallelic pathogenic variants in the HEXB gene, characterized by GM2 ganglioside accumulation in the nervous system and progressive central nervous system degeneration.
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Sandhoff disease, adult form 1 trial
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Sandhoff disease, infantile form 0 trials
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Sandhoff disease, juvenile form 0 trials