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The CTSF began funding a GM2 Inhibitor study being conducted at Boston College in January 2016. The study has two parts - testing the substrate known as 3f alone and in combination with the ketogenic diet. The first part of the study has been complete, testing the 3f alone and the results showed a 13% reduction in GM2 - which is significant!! You can see the progress report by clicking the link above.
The second phase of the study has been started incorporating the ketogenic diet. We will post those results when the work is done. This inhibitor substrate (3f) is the property of the University of Michigan so they control its rights and pace to market. Our goal is only to determine if the drug would be a useful treatment for Tay-Sachs. While the inhibitor is not a cure - it does appear to be a promising tool to slow the pace of the disease as we search for a better option.
This update will be posted on the Cure Tay-Sachs website under Current Research Updates. If you have any questions or comments about this update I can be reached at firstname.lastname@example.org or you can call the foundation offices at (216) 812-5855. More updates will be posted as they become available.
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