NTSAD Research: Developing Treatments, Therapies, and a Cure

The National Tay-Sachs & Allied Diseases Association (NTSAD) is dedicated to finding effective treatments and – ultimately – a cure for Tay-Sachs, Canavan, GM1, and Sandhoff diseases.

Working with our leadership, we drive research, forge collaboration, and foster community among researchers around the world. NTSAD research is informed by our Scientific Advisory Council (SAC), which is made up of leading experts in leukodystrophyA group of rare genetic disorders that affect the central nervous system by disrupting the growth or maintenance of the myelin sheath that insulates nerve cells. Myelin is commonly referred to as the brain's "white matter", myelin covers nerve cells and ensures the clear transmission of nerve impulses from one part of the body to another. These disorders are progressive, meaning that they tend to get worse throughout the life of the patient. The word leukodystrophy comes from the Greek words leuko (meaning white), trophy (meaning growth), and dys (meaning ill). If you put these words together, the word leukodystrophy describes a set of diseases that affect the growth or maintenance of the white matter (myelin). More and lysosomal storage disorders. Our Research Initiative has awarded more than 60 grants and provided more than $4 million in funding since 2022.

Additionally, we support individuals and families to interpret scientific literature and take part in clinical trials and other vital research initiatives.

For information on a specific disease, please visit our Diseases page.