The Research Update Small Group Discussions gave families the opportunity to speak directly with leading scientists searching for a cure, including Tay-Sachs Gene Therapy Consortium researcher Miguel Sena-Esteves, PhD. Everyone was eager to learn when a gene therapy clinical trial might begin and what funding is needed. NTSAD's commitment to finding a cure has not wavered and will continue to partner with the Cure Tay-Sachs Foundation to fund this important research that is a top priority for all of us.
Additionally, we continue our support of other potential therapies. During the session we also heard from Brett Crawford, PhD on substrate inhibitors and the importance of looking broadly at Tay-Sachs and related diseases to more quickly find a cure; Brandon Wustman, PhD on the possible connections between Alzheimer's Disease and Tay-Sachs; and Evan Snyder, MD, PhD on stem cells.
We have an incredible Scientific Advisory Board and know that we are working with the best researchers in the field, who are asking the right questions and designing innovative methodologies to answer them.
We are incredibly proud of the investments we are making across the board and we are so very grateful for all of your support that helps make this research possible. Hope IS on the horizon.
Being in San Diego under the blue skies and warm sunshine I had to be dragged to the Research Session by Carol Zimmerman and held there by Rose Urban, as I really wanted to get some Vitamin D. Â However, once it began I was really glad I stayed. Â Gone are the long, boring, PowerPoint days of presentations and I found myself interested and engaged with the presenters. Â I learned a lot, and found myself grateful that there are so many dedicating their lives to finding a cure. ~ Emilyâ€™s mom Karla Jones
Even though I know that results will probably never help my child, I am so grateful that all of these people are trying to find cures & preventions for these horrific diseases. ~ Overhead during the session
The research session was fantastic! It was held in an informal environment where interaction with the professionals in the medical area was easy, and very informative! I learned a lot about Tay-Sachs and got a glimpse as to where the research in this area is heading! ~ Cary Berman, LOTS