Donations by Mail or Phone:
(617) 277-4463
Checks payable to NTSAD Heringer Fund
2001 Beacon Street, Suite 204
Boston, MA 02135
The Heringer Family Fund for Research has been raising awareness and funds in search of effective treatments and a cure for Tay-Sachs disease by supporting cutting-edge research to help eradicate rare, genetic diseases.
Tay-Sachs is a rare, genetic disorder caused by the absence of beta-hexosaminidase (HexA) causing various neurological symptoms, impacting speech and mobility. There are three forms of Tay-Sachs: Infantile, Juvenile, and Late Onset Tay-Sachs. It’s most commonly seen in infants and older children, who experience the most severe forms and usually do not live past childhood. Late Onset Tay-Sachs is a progressive form that affects individuals slowly over time, but is not fatal.
Together, we have raised more than $60,000 for research through NTSAD and your support has made a difference.
- Patients with Late Onset Tay-Sachs disease are currently participating in a clinical trial at the National Institutes of Health (NIH), at sites in Atlanta, Boston, Los Angeles, and New York, as well as several other countries around the world, for a drug developed by Sanofi Genzyme that hopes to arrest the progression of the disease. It is a multinational, double blind, placebo-controlled study.
- The Jacob Sheep project supports the research, care, and treatment of a rare breed of sheep, which are born with a natural form of Tay-Sachs. The Jacob Sheep have been treated with gene therapy with viable success. In other words, the Jacob Sheep, a large animal model for gene therapy trials, have directly led to early-stage planning of a possible AAV clinical trial for Late Onset of Tay-Sachs and Sandhoff (LOTSS) diseases in patients!
- As of December 2021, there are ten active clinical trials across all four diseases served by NTSAD (Tay-Sachs, Canavan, GM1 Gangliosidosis, and Sandhoff). Research and therapies in these diseases benefit our entire community, and many other allied diseases, such as Alzheimer’s and Parkinson’s.
- In January 2021 members of NTSAD’s LOTSS Community participated in a “Listening Session” with the Food and Drug Administration (FDA), where patients shared their experiences and recommendations for the FDA’s process to approve and evaluate clinical trials to help accelerate the process, with consideration for specific concerns of the rare disease population.
- The 4th Annual Late Onset Think Tank held virtually in October fostered collaboration among elite doctors, scientists, and experts in the field of genetics and rare diseases to share information and research developments as well as to generate new ideas and innovation.
- Finally, NTSAD has hired its first ever Research Director, Valerie Greger, Ph.D. and welcomes Kathleen M. Flynn as the organization’s CEO who succeeds Sue Kahn.
We are grateful to the ongoing generosity of our family and friends, which means so much more to us than words can express.
Hi Family and Friends,
I recently learned that I am affected with Late Onset Tay-Sachs.
I was nervous to share my story because I didn’t want people to look at me differently. However, it’s more important to help raise awareness and be an advocate for this rare disease. It’s crucial to me to speak for others who feel they haven’t been heard, as they are impacted by something beyond their control.
I realize that this disease is a part of me, but it doesn’t define me.
Love,
Becca
Learn more about my personal story featured in Women’s Health Magazine here.
Please Donate Today
Donations by Mail or Phone:
(617) 277-4463
Checks payable to NTSAD Heringer Fund
2001 Beacon Street, Suite 204
Boston, MA 02135
To set up annual recurring donations, please contact us at (617) 277-4463.
With your help, Tay-Sachs researchers can put in motion clinical trials for treatment and cure.
Read more about Tay-Sachs here.