Discovering Effective Treatments & A Cure for Fragile X Syndrome

Your support enables FRAXA Research Foundation to fund groundbreaking research and life-changing clinical trials.


FRAXA's Impact to Date

Read our report for an update on Fragile X research and progress towards a cure.

Explore 29 years of outstanding Fragile X research.


Direct Investment in Fragile X Research


Teams Actively Researching Fragile X Syndrome


Pharmaceutical and Biomedical Partners


Fragile X Research Grants Awarded

How Does FRAXA Help?

FRAXA’s mission is to find effective treatments and ultimately a cure for Fragile X syndrome. We directly fund research grants and fellowships at top universities around the world. We partner with biomedical and pharmaceutical companies, large and small, to bridge the gap between research discoveries and actual treatments.

Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders.

What is Fragile X Syndrome?

Fragile X syndrome (FXS) is the most common inherited cause of autism and intellectual disabilities. It affects 1 in 4,000 boys and 1 in 8,000 girls worldwide.

Fragile X syndrome occurs when a single gene on the X chromosome shuts down. This gene makes a protein needed for normal brain development. In FXS it does not work properly, the protein is not made, and the brain does not develop as it should.

The Latest from FRAXA

Your Support on Giving Tuesday 2023 is Transforming Research

November 29, 2023

On Giving Tuesday 2023, the generosity of 191 donors raised $333,517 for FRAXA Research Foundation, advancing research and hope for a cure for Fragile X syndrome.

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An Update from Harmony Biosciences on Giving Tuesday

November 28, 2023

Harmony Biosciences recently completed its acquisition of Zynerba Pharmaceuticals. I am excited for us to progress their work in pursuit of a treatment option for people living with Fragile X syndrome.

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Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

November 7, 2023

FRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.

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Virtual Research Q&A with Mike Tranfaglia, MD, and Katie Clapp

November 2, 2023

Please join FRAXA co-founders, Katie Clapp and Dr. Michael Tranfaglia, for a Research Q&A via Zoom on Wednesday, November 15, 2023 at 12:00 pm (noon) ET

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Recruiting: Tetra Therapeutics Initiates Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome

October 22, 2023

Tetra Therapeutics has launched large scale clinical trials of their phosphodiesterase (PDE) inhibitor for males ages 12-45 with Fragile X syndrome. FRAXA Research Foundation’s basic and translational research pointed the way to phosphodiesterase inhibitors to treat Fragile X.

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C-subunit Mitochondrial Leak Channel in Fragile X Syndrome

September 20, 2023

Explore Yale’s groundbreaking study on mitochondrial leak channels, set to revolutionize Fragile X syndrome treatment. Funded by a $100,000 FRAXA grant.

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