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GM2 gangliodoses are a subgroup of lysosomal storage disorders and consist of three closely-related genetic conditions. Tay-Sachs disease is caused by mutations in the HEXA gene, Sandhoff disease is caused by mutations in the HEXB gene and the AB variant is caused by mutations in the GM2A gene. The most common form of all three presents in infancy and often causes death by age five. Symptoms typically begin between 2-10 years of age in individuals with the juvenile-onset form, and individuals with the adult-onset form may begin showing symptoms in their 20s or 30s. Management of the disease is based on symptoms and is often supportive, as there are currently no approved treatments for GM2.
The latest on our research
We are jumpstarting our GM2 program to accelerate research for the condition. We welcome all patients and caregivers interested in participating and spreading the word to the GM2 community.