The Cure Angelman Now (CAN) campaign is an annual fundraising effort led by parents, family and friends of individuals living with Angelman syndrome to help raise funds to support and advance critical research toward our mission of curing Angelman syndrome. Earlier this month, our top 2023 CAN fundraisers were invited to Texas A&M University where they toured the lab of Dr. Scott Dindot to see the impact of their generosity in action.
Dr. Scott Dindot has invested over twenty years in Angelman syndrome research. He is a pioneer in the field and a long-time partner of FAST. His relentless pursuit of discovering how to “stop the stop” and activate the paternal copy of UBE3A (pillar 2) has led to the discovery of GTX-102, an investigational ASO currently in a clinical trial under the sponsorship of Ultragenyx Pharmaceuticals.
Dr. Dindot and his lab also created the first large animal model of Angelman syndrome using the pig. And on this trip, FAST donors, board members and staff got the opportunity to tour multiple areas of Dindot’s lab and for the first time, got to meet the Angelman syndrome pig (and its litter), funded by FAST.
Thank you to everyone in our community who donates, fundraises, sponsors and solicits support of critical research like this. It’s the collective effort from people near and far that is helping to build the pipeline of investigational therapeutics with the potential to change the lives of individuals living with Angelman syndrome. We are so grateful for your support of our mission!