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

Researchers analyzing auditory system data to study therapeutic targets for Fragile X syndrome.

Exploring the Auditory System Therapeutic Target for Fragile X Syndrome

November 20, 2025

Auditory system therapeutic target for Fragile X syndrome research uses a rat model to connect excess protein synthesis with disrupted circuits and auditory hypersensitivity.

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Dr. Emily Osterweil and her research team at Harvard Medical School studying Fragile X syndrome using human-derived brain models

Identifying New Therapeutic Approaches Using a Human Fragile X Model

November 12, 2025

Human Fragile X syndrome model research identifies new therapeutic approaches by revealing how FMRP loss affects human neurons and brain circuits.

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Nicola Elvassore and his research team at the University of Padua, Italy, in their laboratory conducting Fragile X brain organoid studies supported by FRAXA Research Foundation

Cell Type-Specific Protein Dysregulation in Fragile X Syndrome Brain Organoids

November 10, 2025

Study finds that brain cell types respond differently to gene silencing, pointing to the need for customized treatment approaches in Fragile X.

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FRAXA's Impact to Date

FRAXA's Interactive Fragile X Timeline

Explore 30 years of outstanding Fragile X research.

$38.2M

Direct Investment in Fragile X Research

49

Teams Actively Researching Fragile X Syndrome

34

Pharmaceutical and Biomedical Partners

659

Fragile X Research Grants Awarded