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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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

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

May 20, 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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Recruiting: Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option

May 20, 2024

Harmony Biosciences is now recruiting for the RECONNECT Phase 3 clinical trial of ZYN002, a potential treatment for Fragile X syndrome. The trial offers an at-home participation option, making it accessible for patients across the US, Australia, and the UK. Join us in this groundbreaking study and contribute to the future of Fragile X syndrome treatment.

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Your Opinion Matters: Help Shape Gene Therapy Research for Fragile X Syndrome

May 8, 2024

Help shape the future of Fragile X syndrome treatment by sharing your thoughts on gene therapy. Your input is vital as we explore potential new treatments that target the genetic cause of FXS. Participate in the Patrick Wild Centre’s survey today and make your voice heard in this important research.

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FRAXA's Impact to Date

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