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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Aripiprazole (Abilify) in the Treatment of People with Fragile X: An Anecdotal Account

The aim of this article is to discuss the use of Abilify (generic name: aripiprazole) as a treatment for people with Fragile X syndrome (FXS). As an “off-label” prescription, Abilify targets behaviors such as irritability, aggression, self-injury and severe tantrums.
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Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases

This is the first in a series of webinars focused on current topics in Fragile X research. In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Programand Fabio C. Tucci, PhD, Chief Operating Officer and co-founder at Epigen Biosciences, Inc.
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Help NIH Understand the Impact of COVID-19 on the Rare Disease Community

The NIH-sponsored Rare Diseases Clinical Research Network (RDCRN) has created this survey for people living with a rare disease in the US. The goal is to better understand the unique impacts of COVID-19 on the rare disease community.
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