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

Targeting Serotonin 1a Receptors to Reverse Neurobehavioral Phenotypes

March 30, 2026

Neurolixis’ new drug targets serotonin 1A receptors (aka 5-HT1A), showing promise in preclinical studies for Fragile X syndrome, funded by a FRAXA grant for future clinical trials.

FRAXA Backs Upcoming Phase 2B Trial of SPG601 for Fragile X Syndrome

March 15, 2026

FRAXA Research Foundation is supporting a Phase 2b clinical trial of SPG601, an experimental therapy targeting BK channels in fragile X syndrome, following promising early results.

Spinogenix and FRAXA Advance SPG601 Into Phase 2b Trial for Fragile X Syndrome

March 2, 2026

Spinogenix and FRAXA are supporting a Phase 2b clinical trial of SPG601 in Fragile X syndrome, building on Phase 2 signals and FDA designations.

FRAXA's Impact to Date

FRAXA's Interactive Fragile X Timeline

Explore 30 years of outstanding Fragile X research.

$38.4M

Direct Investment in Fragile X Research

37

Teams Actively Researching Fragile X Syndrome

34

Pharmaceutical and Biomedical Partners

696

Fragile X Research Grants Awarded