Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

2017 Seventh Annual Day of Hope: Ideas

If one of these ideas, or a it's combination of a few, or if you have an idea of your own, contact Becky at This email address is being protected from spambots. You need JavaScript enabled to view it.. We'll get you all set up and help you from A-Z!

On Facebook

Watch the DAY OF HOPE page on Facebook for announcements! And be sure to post your event in the group, too! We can all encourage each other and share ideas and celebrate success!


Need Ideas?

Click HERE for inspiration to be a #rareGENEius and be part of the Day of Hope!

Together. We are strong.

We will make a difference!

We are making a difference!

What's New?

In May 2017, NTSAD awarded $200,000 in grants to:

Alessandra Biffi, MD, Director of Gene Therapy at Dana-Farber, for her work to test a novel gene therapy approach for Tay-Sachs and Sandhoff. Her work has significant potential to achieve transformative outcomes due to her expertise and well-developed research plan.

Heather Gray-Edwards, PhD, Scott-Ritchie Research Center at Auburn University, to allow her to evaluate the efficacy of the state-of-the-art gene therapy in the Tay-Sachs sheep. (Read more about Jacob's Sheep here.) Her work and the resulting data will contribute to future clinical trials in patients.

Stephane Demotz, PhD, Dorphan, and Tim Wood, PhD, Greenwood Genetic Center, to analyze a sugar molecule as a biomarker for GM1, and allow them to explore the optimal treatment conditions of an investigational pharmacological chaperone drug candidate for GM1.

Read more about the grants in May's Research Review here.