Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives

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.

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Yue Feng, PhD

Functional Interplay Between FMRP and CDK5 Signaling

FRAXA-funded work showed CDK5 signaling is disrupted in Fragile X. CDK5 drugs are in development for Alzheimer’s so this pathway offers a promising new FX treatment angle.

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FMRP-MAP1b RNA Interactions in Fragile X Syndrome

FRAXA-funded research by Dr. Mihaela Mihailescu at Duquesne University revealed insights into FMRP and RNA structure in Fragile X. Results were published.

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Jay Brenman, PhD, at University of North Carolina, FRAXA research grant

Dendritic Spine Formation and Fragile X

With $35K from FRAXA, Dr. Jay Brenman at UNC used fruit fly models to study Fragile X and uncover key disease mechanisms.

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FRAXA Funded Research

Current Research Grants (40)