Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Natural history studies document the progression of the disease. The more data there is about the progression of the disease from medical data and patient experience, the clearer the endpoints are to determine whether a therapy is working or not. The PIN is a format to help families contribute this information. Therefore, participating in the PIN and natural history studies is important and critical.

Join the GM2 Tay-Sachs & Sandhoff PIN here or the GM1 PIN here as soon as you can!