Finding effective treatments and ultimately

A CURE FOR FRAGILE X SYNDROME

FRAXA's Impact to Date

$
0
27,674,500

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
32

TEAMS ACTIVELY RESEARCHING FRAGILE X

Current Research Grants
0
3

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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Correcting Sensory Processing in Fragile X Mice by Modulating Kv3.1

FRAXA has awarded a $90,000 grant to Carlos Portera-Cailliau, PhD and Nazim Kourdougli, PhD at UCLA to investigate whether a novel drug can rescue sensory processing deficits in Fragile X mice. People with Fragile X have similar problems in sensory processing. This new drug acts on Kv3.1, a promising Fragile X treatment target also being pursued by UK-based Autifony Therapeutics based on FRAXA-funded research done at Yale.
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A Day in the Lab with FRAXA Investigator Dr. Tue Banke

Recently Laurie Bowler and her 19-year-old son Casey, who has Fragile X syndrome, visited FRAXA research grant recipient Dr. Tue Banke at his University of Washington laboratory. We hope you enjoy Laurie's wonderful description of their adventure! FRAXA awarded $90,000 to Dr. Banke to study the Developmental Profile of Glutamatergic Synapses in Fragile X.
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Gene Therapy Translational Studies for Fragile X Syndrome

With a $90,000 award from FRAXA Research Foundation, Drs. Ernest Pedapati, Christina Gross, and student Lindsay Beasley will pursue preclinical gene therapy approaches for treating Fragile X syndrome at Cincinnati Children’s Hospital.
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