Slide Slide Slide Finding effective treatments and ultimately
A CURE FOR FRAGILE X SYNDROME
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FRAXA's Progress
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FRAXA's Impact to Date

$
0
30,692,922

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
29

TEAMS ACTIVELY RESEARCHING FRAGILE X

Current Research Grants
0
4

GENE THERAPY AND PROTEIN REPLACEMENT STUDIES

Current Studies
0
31

PHARMACEUTICAL AND BIOMEDICAL PARTNERS

0
18

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 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.

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

View all News & Updates

Results: Clinical Study of Non-Invasive EEG for Children Ages 2-7

Dr. Carol Wilkinson, MD PhD, and Dr. Charles Nelson, PhD, at Boston Children’s Hospital are recruiting children ages 2-7 years with Fragile X syndrome to join a study of brain differences using non-invasive EEG.
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Educating Young Children with Fragile X Syndrome

When a team of elementary school teachers and therapists asked us to share strategies for working with young children who have Fragile X syndrome, we developed this session featuring tips, techniques, and stories. Katie Clapp, co-founder of FRAXA Research Foundation and parent of a young man with Fragile X, and Tracy Antonelli, whose two teenage boys have Fragile X, present this session to help teachers assist their students.
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Parkinson’s Therapy May Hold Promise for Fragile X

A study funded by FRAXA in Italy has encouraging results for people with Fragile X: drugs that block adenosine receptors (A2A) reversed signs of Fragile X in a mouse model. "One of the most intriguing things about this study is that it points to an entire drug class (not just the one drug used) as potentially therapeutic for Fragile X. Many available compounds block A2A receptors, and we know they are safe and effective.
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The Why and How of FRAXA’s Work to Find a Cure for Fragile X

Thank you Talk Fragile X for having FRAXA's cofounder Katie Clapp as a guest on your podcast! It was a pleasure to share why FRAXA got started and our motivation for finding effective treatments and ultimately a cure for Fragile X syndrome.
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Beneath the Surface of Fragile X Syndrome: Study Sheds Light on What’s Happening in Nerve Cells

This FRAXA-funded project has turned up some surprising results. At first, it might seem Kurosaki and Maquat have found yet another cellular process which is malfunctioning in Fragile X. But this finding is intimately related to previous findings of abnormal protein synthesis and misregulated transcription in Fragile X. FMRP (the protein lacking in Fragile X syndrome) is involved in chaperoning messenger RNAs within cells to active sites, and in controlling their translation into many different proteins. Some of these proteins are transcription factors, which feed back to the nucleus to control gene expression.
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COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

The two COVID-19 vaccines that recently received emergency approval from the US and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies.
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