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

Research on GM1 Gangliosidosis-1

Research is making exciting advances towards a treatment and a cure for GM1.

Research Updates

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Types of Research

It is important to note that research on Tay-Sachs and Sandhoff diseases is also meaningful to GM1 as these diseases share many biochemical traits. Below is an overview of the types of research with links to Research section for more details.

Gene Therapy

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. Gene Therapy

Molecular Chaperone

Pyrimethamine (PYR) molecular chaperone is currently in clinical trials of adult Late Onset Tay-Sachs. At the right dose PYR could increases enzyme activity. Pharmacological or Molecular Chaperone Therapy

Substrate Inhibition

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. Substrate Inhibition

Stem Cells

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. Stem Cell Therapy

Bone Marrow Transplant

Bone Marrow Transplant is an extremely invasive and experimental intervention. Bone Marrow Transplant

Courtesy of the American Society of Gene and Cell Therapy. Learn more here.