Nervous System Home > Tay-Sachs Research

Tay-Sachs research studies are currently investigating more effective treatment methods and attempting to find a cure for the condition. Current areas of focus include enzyme replacement therapy, bone marrow transplant, and gene therapy. Volunteers who participate in Tay-Sachs research studies have the first chance to benefit from treatments that have shown promise in earlier research.

Tay-Sachs Research: An Overview

Tay-Sachs research studies are designed to answer important questions about the disease and to find out whether new approaches are safe and effective. This research already has led to many advances, and researchers continue to look for more effective methods for dealing with Tay-Sachs.

Current Areas of Focus of Tay-Sachs Research

There is no effective treatment for Tay-Sachs disease. However, researchers are pursuing several approaches to finding a Tay-Sachs cure.
Scientists are exploring enzyme replacement therapy to provide the hexosaminidase-A (HEXA) that is lacking in babies with Tay-Sachs. Bone marrow transplantation has been attempted also, but to date has not been successful in reversing or slowing down damage to the central nervous system in babies with Tay-Sachs. Another avenue of Tay-Sachs research is gene therapy, in which scientists transfer a normal gene into cells to replace an abnormal gene. This approach holds great promise for curing Tay-Sachs.

Potential Benefits of Participation in Tay-Sachs Research

In order for some Tay-Sachs research to be conducted, volunteers are needed. Patients who join Tay-Sachs research studies have the first chance to benefit from treatments that have shown promise in earlier research. They also make an important contribution to medical science by helping doctors learn more about the disease. Although Tay-Sachs research trials may pose some risks, researchers take careful steps to protect their patients.
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Last reviewed by: Arthur Schoenstadt, MD
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