Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

Tay Sachs is a devastating genetic disease with no known cure. But now, experts are hoping a new push for awareness could help parents down the road.

Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life. Scientists have reasoned that replacing the ...

A Florida state legislator sponsored the nation's first state-backed genetic screening program after his son died from a rare genetic disease.

After losing his son Drew to Tay-Sachs Disease in 2019, Adam Anderson’s life took on a new path. He ran for local office in Florida and became a leader in rare disease policy, creating a new genetic ...

Zeng, B. J., Torres, P. A., Viner, Tabitha, Wang, Z. H., Raghavan, S. S., Alroy, J., Pastores, G. M., and Kolodny, E. H. 2008. "Spontaneous Appearance of Tay-Sachs ...

Early genetic counseling gives families crucial information and support, turning uncertainty about rare diseases into informed decisions, community connections and hope for their children’s health and ...