Finding effective treatments and ultimately

A CURE FOR FRAGILE X SYNDROME

FRAXA's Impact to Date

$
0
27,222,846

Direct Investment in Fragile X Research

Current FRAXA Funded Fragile X Research

0
4

ONGOING FRAGILE X CLINICAL TRIALS AND STUDIES

Current Clinical Trials
0
30

TEAMS ACTIVELY RESEARCHING FRAGILE X

Current Research Grants
0
4

GENE THERAPY AND PROTEIN REPLACEMENT STUDIES

Current Studies
0
28

PHARMACEUTICAL AND BIOMEDICAL PARTNERS

0
11

COUNTRIES ARE HOME TO RESEARCH TEAMS

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 6,000 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.

Learn more about Fragile X syndrome

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.

Donate to FRAXA's mission

Latest Fragile X News & Updates

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One Person Can Make a Difference

When Ellen Skala, also referred to as Elle, contacted us in August 2018 about creating a personal fundraising campaign for FRAXA, we were excited about her interest in supporting our mission. Elle set a goal to raise $10,000 for Fragile X research by her birthday, and she committed to shaving her head if she met her fundraising goal!
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Ganaxolone Fragile X Clinical Trial Showed Disappointing Results

Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.
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FRAXA Plans for Fragile X Research: 2019 Funding Opportunities

Each year, FRAXA funds over $1 million in research aimed at finding new and improved treatments -- and ultimately a cure -- for Fragile X syndrome. The application deadline for FRAXA Fellowships is February 1, and new fellowships are announced in early March. Clinical trial proposals are accepted anytime. Our current priority is clinical trials.
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