Our goal is to find effective treatments and—ultimately—a cure for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases.
To achieve this goal, NTSAD formed a Research Task Force comprised of members from our Scientific Advisory Council (SAC) and other experts in lysosomal and 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 disorders. Together, they identified four key research areas on which to focus. These are the pillars of our research priorities.