Discovering Effective Treatments & A Cure for Fragile X Syndrome

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

A young man with Fragile X syndrome smiling while sitting on the floor at home.

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.

Every member of our staff and Board of Directors is a parent of one or more children with Fragile X, making our commitment to this cause deeply personal.

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.

Symptoms of Fragile X syndrome can include developmental delays, social anxiety, and learning disabilities, which vary between individuals.

The Latest from FRAXA

Dr. Anirudh Acharya and Dr. Nien-Pei Tsai study mitochondrial function in a Fragile X syndrome research laboratory.

Pharmacological Activation of PGC-1α to Treat Fragile X Syndrome

December 22, 2025

Dr. Tsai and Dr. Acharya are testing whether boosting mitochondrial function can improve key Fragile X syndrome features in a mouse model and inform treatment strategies.

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Group photo of Callum Cup IX participants gathered at Millburn High School during the annual charity soccer match supporting Fragile X research.

Callum Cup IX Brings Community Together, Raising $20,000 for Fragile X Research

December 17, 2025

Callum Cup IX brought the Millburn FC community together, raising $20,000 for the FRAXA Research Foundation in support of Fragile X research.

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Drs. Oswald Steward and Patricia Pirbhoy in their laboratory, studying RNA localization and gene activity in Fragile X syndrome using spatial transcriptomics.

Uncovering the Dynamics of FMR1 Using Spatial Transcriptomics for Fragile X

December 12, 2025

Scientists investigate how changes in RNA localization affect cognition and behavior in Fragile X, building knowledge toward more precise therapies.

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FRAXA's Impact to Date

FRAXA's Interactive Fragile X Timeline

Explore 30 years of outstanding Fragile X research.

$38.3M

Direct Investment in Fragile X Research

52

Teams Actively Researching Fragile X Syndrome

34

Pharmaceutical and Biomedical Partners

695

Fragile X Research Grants Awarded