Working toward a Cure
FRAXA’s mission is to find effective treatments and a cure for all children and adults with Fragile X, by directly funding the most promising research. As the pace of Fragile X research accelerates, the prospects of finding effective treatments and a cure for Fragile X and autism continue to improve. FRAXA-funded researchers at universities around the world are leading the way.
FRAXA’s research portfolio spans the spectrum of basic science, pre-clinical, and clinical research — all coordinated to make the most of each dollar. We maintain a diversified approach, developing several treatment strategies in parallel, since success is never certain in developing a single drug. We continue to fund research to define the precise defect in the Fragile X brain, because these basic studies may yield additional important therapeutic targets.
Where are we now?
In the words of FRAXA Scientific Advisor Dr. Justin Fallon, “Fragile X is poised to become a triumph for translational research and the design of rational therapeutics for brain disease.”