Your Donation Matters!
FAST’s scientists know the underlying cause of Angelman syndrome. A single nonfunctional or missing gene. They have reversed the symptoms of the disorder in mice and rats using multiple strategies. A cure is no longer a matter of if, but when.
To this end, FAST is driving translational research and supporting the advancement of therapeutics for animal models to humans living with Angelman syndrome. This includes not only supporting 9 different disease of gene-modifying therapeutic strategies, but also exploring the benefits of downstream drug targets, while working rigorously to create the foundation for human clinical studies. Through robust investments in clinical endpoints and biomarkers, the development and characterization of animal models, and supporting novel tools for platform testing of different therapeutic candidates, FAST is deeply invested in bringing promising interventions to clinical trials. We live each day with the goal to bring practical treatments for every individual living with Angelman syndrome into current medical practice, as safely and quickly as possible, and we will not stop until that is achieved.