Disease

Sandhoff Disease

About the Disease
Sandhoff Disease, also known as total hexosaminidase deficiency, is related to gm1-gangliosidosis, type i and gm2-gangliosidosis, ab variant. An important gene associated with Sandhoff Disease is HEXB (Hexosaminidase Subunit Beta), and among its related pathways/superpathways are Metabolism and Sphingolipid metabolism. The drugs Miglustat and Anti-HIV Agents have been mentioned in the context of this disorder. Affiliated tissues include spinal cord, eye and brain, and related phenotypes are macrocephaly and failure to thrive

Common Targets
GBA1 | UGCG | G6774 | HEXA | Chaperone (nonspecified subtype) | HEXB | OGA | GBA2

疾病靶点研报
Sandhoff Disease

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