Drs. Oostra, Warren, and Nelson discovered the Fragile X gene and its FRAXA mutation in 1991.

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.

Our Strategy

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.”

What is the research most likely to lead to treatments for Fragile X in the near future? While no one can foresee the future, we are optimistic that the mGluR Theory has tremendous promise.
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Clinical trials are in progress, and some are seeking new participants. We hope these trials will be successful for the sake of all affected by Fragile X.
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Look here for latest news about Fragile X.
Treatment advances, key discoveries, Fragile X family stories, and FRAXA in the news.
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The pace of Fragile X research is accelerating, and prospects of finding treatments and a cure for Fragile X and autism continue to improve. FRAXA research is leading the way.
Read More