Discovering Effective Treatments & A Cure for Fragile X Syndrome

Your support enables FRAXA Research Foundation to fund groundbreaking research and life-changing clinical trials.

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FRAXA's Impact to Date

Read our report for an update on Fragile X research and progress towards a cure.

Explore 30 years of outstanding Fragile X research.

$34.8M

Direct Investment in Fragile X Research

32

Teams Actively Researching Fragile X Syndrome

33

Pharmaceutical and Biomedical Partners

641

Fragile X Research Grants Awarded

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.

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.

The Latest from FRAXA

We Started Out Small, Now Look…

May 13, 2021

FRAXA Research Foundation is spreading awareness across the world! We’d love to see you light up our world on July 22 as part of World Fragile X Day!

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NPR Feature – A Fragile X Treatment May Be On The Horizon

May 7, 2021

With a promising new treatment on the horizon, NPR reflects back at the past 10 years, providing a glimpse into both the science and research of Fragile X through the eyes of key researchers, in addition to the lives of this family, their quest for a cure, and what that may look like for their son, Andy.

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FRAXA-DVI laboratory

FRAXA Drug Validation Initiative (FRAXA-DVI)

May 6, 2021

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS).

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Pharmacotherapeutic Effects of Cannabidiol (CBD) in Fragile X syndrome (FXS) and Autism Spectrum disorder (ASD)

May 6, 2021

This study will test CBD (cannabidiol) treatment in male and female Fragile X mice to learn how and why it works and whether gender affects responses to CDB treatment. Along with clinical trials, this study will help us to understand and optimize the potential of CBD as a behavior-regulating treatment for Fragile X.

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NPR Short Wave, “A Fragile X Treatment May Be On The Horizon”

May 6, 2021

Katie Clapp and Michael Tranfaglia’s son was born with a genetic disorder that affects brain development. It makes it hard to learn language and basic daily tasks and often is accompanied by a host of other disorders. To help find a cure, they started a foundation and raised research money. After se…

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Holly Roos Joins the FRAXA Team!

May 5, 2021

FRAXA Research Foundation is excited that long time Fragile X advocate and well-known figure within the Fragile X community, Holly Roos, is joining our team as Community Services Director. We are thrilled with the energy and enthusiasm Holly brings to the team.

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