Finding effective treatments and ultimately

A CURE FOR FRAGILE X SYNDROME

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FRAXA's Impact to Date

$
0
28,126,000

Direct Investment in Fragile X Research

Current FRAXA Funded Fragile X Research

0
6

ONGOING FRAGILE X CLINICAL TRIALS AND STUDIES

Current Clinical Trials
0
33

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.

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Latest Fragile X News & Updates

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FRAXA is Thinking About You

We recognize that with the constantly changing COVID-19 situation around the world, this is an unprecedented time for everyone — a time that, for many, is filled with uncertainty. Like many of you, we have children or adults at home with Fragile X syndrome. We truly understand what a great challenge this can be and are here to lend a hand.
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Companies Move to Advance Potential Cognitive Treatment for Fragile X

Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits, including Fragile X syndrome and Alzheimer’s disease. The agreement builds on an earlier collaboration between the two companies, and aims to further accelerate BPN14770’s development and potential marketing. It is currently in clinical testing in both Fragile X and Alzheimer’s patients.
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Ketogenic Diet Eases Symptoms in Fragile X Male Mice

The Westmark laboratory continues to study sleep and rest-activity cycles in Fragile X mice as a potential outcome measure that correlates between preclinical and clinical research. The analysis of sleep EEG in the mice has proven more labor intensive than they anticipated, but the team is collaborating with Dr. Rama Maganti’s laboratory at UW-Madison on the development of computer scrips to speed up the analysis.
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