Discovering Effective Treatments & A Cure for Fragile X Syndrome

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

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FRAXA's Impact to Date

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

Explore 30 years of outstanding Fragile X research.

$34.8M

Direct Investment in Fragile X Research

32

Teams Actively Researching Fragile X Syndrome

33

Pharmaceutical and Biomedical Partners

641

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

Renewed Hope: Navigating Towards a Cure for Fragile X Syndrome

April 16, 2024

Discover how Dr. Peter Todd’s latest Fragile X Syndrome research offers hope for advanced treatments and a possible cure, marking a new era in FXS therapy.

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Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments

March 13, 2024

Explore how the FRAXA Drug Validation Initiative is revolutionizing Fragile X syndrome treatment, leading the charge towards innovative therapies and hope for affected families.

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Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome

March 7, 2024

Join Dr. Tsai and Dr. Kumar on a journey into novel treatment avenues for Fragile X syndrome. Learn how activating mGluR7 could be a game-changer, opening up uncharted therapeutic territory.

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Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice

March 6, 2024

Treating Fragile X syndrome will likely require a combination of drugs, as a single medication may not address all symptoms. At FRAXA-DVI, Dr. Patricia Cogram and her team recently tested a combination of two investigational new drugs in Fragile X mice, with support from Healx. Together ibudilast and gaboxadol rescued a wide array of symptoms in the mice.

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

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

February 29, 2024

Tetra Therapeutics has launched large scale clinical trials of their phosphodiesterase (PDE) inhibitor for males ages 9-45 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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Celebrating Teamwork: FRAXA’s 7th Year at Bruins 50/50 Event

February 7, 2024

Celebrating FRAXA’s 7th year at Bruins 50/50 Event: teamwork and community unite for Fragile X research.

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