Tay-Sachs and Sandhoff Disease (GM2)

Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis (GM2), a group of rare fatal genetic disorders that progressively damage nerve cells primarily in the brain and spinal cord. Learn how gene therapy is being researched as a potential one-time treatment that could slow or stop disease progression for both diseases.

The subtitling of these videos was funded in part by a donation from Pfizer, Inc.