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

2017 Seventh Annual Day of Hope

What is a Day of Hope?
It is a time for our rare and mighty community to come together to make a difference and show that there is always hope.

All proceeds go to the research that NTSAD funds including the work leading to clinical trials. We're getting closer and together we can get to treatments faster!

Our goal for 2017?

60 events to raise at least $60,000! Let's do it! If you want to participate, contact Becky Benson This email address is being protected from spambots. You need JavaScript enabled to view it.

How can you be part of a Day of Hope?

There are many ways!

  • Be a #rareGENEius.
  • Spread awareness about being the risks of being a carrier.
  • Share your family story and how being rare has impacted your family.
  • Host a lemonade stand in your neighborhood.
  • Create your own giving page and encourage friends to make it go viral for what is important to you.
  • Be a #rareGENEius and check out some of the ideas (in the box to the right), or come up with an idea of your own, and let us know by June 30th and we'll order the fun swag!
  • Whatever you do, be sure to post and tag your photos on Facebook, Twitter or Instagram with #rareGENEius #NTSAD #2017DayofHope.

Let's Rally & Raise Funds for Research!

Let's Make Treatments and a Cure Happen. Now!

2017 Day of Hope Events

2017 Day of Hope

Why Day of Hope?

Why is Day of Hope important?

It is a tough time for federal funding of research in the field of lysosomal storage and leukodystrophy disorders, so researchers need support from organizations and foundations like NTSAD, Cure Tay-Sachs Foundation and Cure GM1. 

This June 18th article from UMass Medical is about one of the projects that NTSAD has been a part of since its inception over 10 years ago.

Read it here.

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.