FRAXA Research Foundation initially partnered with Healx in 2016 to identify existing drugs with potential to treat fragile X syndrome, using machine learning algorithms and computational biology. The study produced results, and now FRAXA and Healx have launched a new round of studies to evaluate combinations of compounds, including both drugs and natural products.
The 18th International Fragile X and Related Neurodevelopmental Disorders Workshop in Quebec, Canada, was a great success, featuring fragile X much more heavily than any previous meeting in this series! We asked our speakers to summarize their work in their own words. These brief updates from researchers investigating fragile X.
FRAXA Research Foundation was founded in 1994 to fund biomedical research aimed at finding a cure for fragile X syndrome and, ultimately, autism. We prioritize translational research with the potential to lead to improved treatments for fragile X in the near term. Our early efforts involved supporting a great deal of basic neuroscience to understand the cause of fragile X. By 1996, these efforts had already begun to yield results useful for drug repurposing. To date, FRAXA has funded well over $25 million in research, with over $3 million of that for repurposing existing drugs for fragile X. Here are some examples of FRAXA-funded work on repurposing available drugs for fragile X syndrome: Lithium In the mid-1990s, the Greenough lab at the University of Illinois discovered that FMRP, the protein missing in fragile X, is rapidly translated in dendrites in response to stimulation of glutamate receptors. FRAXA funded preclinical validation of this discovery in theRead more