The Tay-Sachs Gene Therapy (TSGT) Consortium clinical trials could begin as early as September 2012 – just 15 months away!!

The investigators, funders and other team members met recently to discuss the possibility of accelerating the timeline. Sleeves were rolled up, everything was put on the table and it was decided that we can do this!

There are challenges still to overcome like funding toxicity and animal studies and vector production. In addition to the NIH grant, $1.2 million dollars is necessary to make the goal of September 2012 a reality. The family foundation affiliates have already pledged 75%, or $900,000! Only an additional $300,000 still to raise! Learn more about how you can help below. Don’t be intimidated by the number. Every little bit will bring us closer to a day free of Tay-Sachs and Sandhoff!

Ten years ago NTSAD recommitted itself to funding cutting-edge research and founded the Research Initiative; 23 grants and $1.9 million later we are closer than ever to a cure. We are proud to work behind-the-scenes of managing the research grant process as well as raising needed dollars, but we tip our hat to our family foundation affiliates for their passion and drive to raise the majority of funds.

Together we can beat these diseases! Please join us in the fight and click here to contribute to HOPE in just 15 months. All donations made through the holiday weekend will go directly to this effort. In addition, donations may also be sent anytime to the office; simply note the gift is for HOPE. All major credit cards are accepted. NTSAD 2001 Beacon Street, Suite 204, Boston, MA 02135

Our Family Foundation Affiliates: Cure Tay-Sachs Foundation, Cameron and Hayden Lord Foundation, Mathew Forbes Romer Foundation,
Pesotchinsky Family Fund, and the NTSAD New York chapter.

NTSAD’s HOPE Fundraising Drive
NTSAD has cultivated and supported research for over half a century. In keeping with this tradition, we invite you to join us and our prestigious family foundation affiliates in moving research forward. Contact Diana Pangonis, Development Manager to learn more about NTSAD’s HOPE fundraising drive.

Remember you can support this effort anytime by making a donation and noting HOPE.

NTSAD Researcher receives 2011 Outstanding Investigator Award from American Society of Gene & Cell Therapy

We are proud to report that Miguel, who is the program manager of the TSGT Consortium and associate professor of neurology at the University of Massachusetts Medical School, has received the 2011 Outstanding New Investigator Award from the American Society of Gene & Cell Therapy (ASGCT) for his contributions to the field of gene and cell therapy.

“It’s rare for an investigator at this stage of his career to have accomplished what Miguel has,” said Guangping Gao, PhD, director of the Gene Therapy Center at University of Massachusetts Medical School. “This award recognizes the novel insights Miguel has contributed to the field of gene therapy and is a reflection of what a rising young star he is.”

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