Can we get to the finish line?

Can we get to the finish line?

Can we get to the finish line? We have reached a critical time at FRAXA Research Foundation. By giving to FRAXA today you can get us closer to our goal of effective treatments and ultimately a cure for Fragile X!

In a marathon, the more qualified runners who enter the race, the more who will cross the finish line. We are excited to report that, thanks to your support, FRAXA has grown the field of Fragile X research and we are closer than ever to success!

Through your donations FRAXA funds millions of dollars of research at labs around the world, attracting brilliant minds to the field of Fragile X research. Many FRAXA-funded postdocs become primary investigators, start their own labs, and bring their own postdocs into the field. FRAXA-funded researchers also take their results to larger funding sources like the National Institutes of Health (NIH) and Department of Defense to acquire additional funding to expand their research.
More runners in the race!

With your support, FRAXA has moved beyond the first generation of clinical trials to newly discovered aspects of Fragile X, spurring the next wave of potential treatments. We are funding trials led by Dr. Craig Erickson, Dr. Elizabeth Berry-Kravis and several more.
More runners in the race!

Your donations support FRAXA’s mouse facility, the FRAXA Drug Validation Initiative (FRAXA-DVI), which enables biotech and pharmaceutical companies to test their drugs for potential to treat Fragile X. FRAXA-DVI provides speedy, cost-effective, objective testing of potential new Fragile X treatments. Some companies who have tested their compounds in FRAXA-DVI’s Fragile X mice have seen such positive results that they are moving forward with drug development!
More runners in the race!

What is the result? We are on the cusp of real treatments. We are now working with 28 companies that are bringing additional dedicated individuals into Fragile X research and we have 4 clinical trials in progress today, because of your support. But research is expensive. Will you help us get these runners to the finish line?

Please help us by making a gift today.

Bringing a Promising Fragile X Clinical Trial to Life — FRAXA’s Impact

FRAXA’s Chief Medical Officer, Dr. Mike Tranfaglia, spends much of his day speaking with biotech and pharmaceutical companies that are in the race to find effective treatments for Fragile X. One such company is Tetra Discovery Partners.

Tetra, a startup in Michigan, has a drug in development for Alzheimer’s Disease. The CEO, Dr. Mark Gurney, contacted FRAXA because he saw a scientific paper co-authored by Dr. Mike which suggested that Tetra’s drug, a PDE4D inhibitor, may also be effective in treating Fragile X. Until now, no PDE4D inhibitors were available for use in Fragile X clinical trials because other drug companies were interested only in larger indications. So FRAXA forged a close collaboration with Tetra.

FRAXA tested the drug at FRAXA-DVI and it showed promising results! PDE4D inhibitors can fix Fragile X- related problems in animal models! Tetra then used this data to launch a phase 2 clinical trial. The trial of 30 adults with Fragile X – funded by FRAXA and Tetra, and run by longtime FRAXA grantee Elizabeth Berry-Kravis – is currently underway at Rush University in Chicago. As Dr. Gurney notes, “Dr. Berry-Kravis’ research gives us hope that our drug may address a core biochemical change in Fragile X patients. The results of our collaborative preclinical studies with FRAXA Research Foundation in the Fragile X mice are very promising.

Your donation dollars made possible the drug validation that led Tetra to a phase 2 clinical trial for individuals with Fragile X. FRAXA is now funding this clinical trial for over $200,000. We still need your support to get to the finish line. Until we find treatments and ultimately a cure, we won’t stop expanding the field! Please give now, and thank you for your ongoing support of FRAXA.

Goal: $750,000 • To Date: $204,265

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If you have questions or requests, please call us at (978) 462-1866 or email Dave Bjork at dbjork@fraxa.org.

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If you are interested in a quarterly or yearly recurring gift or have other questions or requests, please call us at (978) 270-3789 or email Dave Bjork at dbjork@fraxa.org.

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