Research is making exciting advances towards a treatment and a cure for Tay-Sachs, Sandhoff and GM-1.
It is important to note that Tay-Sachs research is meaningful to both Sandhoff and GM-1 as these diseases share many biochemical traits.
Visit the Research section for more information on the following:
Researchers have cured Tay-Sachs mice and greatly improved the condition of Tay-Sachs cats. Plans are currently underway to approach the FDA for approval of a Phase I clinical trial. More…
Pyrimethamine (PYR) molecular chaperone is currently in clinical trials of LOTS. At the right dose PYR could increases
enzyme activity. More…
The Zavesca
substrate inhibition clinical trial was not considered a success and it is not currently being pursued as a treatment, but the drug is available for off-label use. Many families felt the drug slowed the disease progression. More…
Exciting breakthrough work on the potential therapeutic uses of stem cells was done in Sandhoff mice. Stem cell therapy is still an emerging field; potential therapies or clinical trials using stem cell
therapy are not immediate. More…
Bone Marrow Transplant is an extremely invasive and experimental intervention. More…
