Discovering Effective Treatments & A Cure for Fragile X Syndrome
Your support enables FRAXA Research Foundation to fund groundbreaking research and life-changing clinical trials.
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.
Every member of our staff and Board of Directors is a parent of one or more children with Fragile X, making our commitment to this cause deeply personal.
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.
Symptoms of Fragile X syndrome can include developmental delays, social anxiety, and learning disabilities, which vary between individuals.
The Latest from FRAXA
Circular RNA Delivery of FMRP in a Human Neurodevelopmental Model of Fragile X Syndrome
FRAXA-funded researchers explore circular RNA to restore FMRP in Fragile X syndrome, working toward a non-viral treatment that could allow repeat dosing.
Recruiting: Mirum Launches Clinical Trial of MRM-3379 for Fragile X Syndrome
Mirum’s MRM-3379 Phase 2 trial builds on FRAXA-funded PDE4D research and is now enrolling males ages 13–45 with Fragile X syndrome. View eligibility and study sites.
Development of Non-Hallucinogenic Substituted Tryptamines (SubTryps) for Fragile X Syndrome
FRAXA researchers are developing DPT-based compounds designed to reduce overactive brain activity without hallucinogenic effects in Fragile X syndrome.
FRAXA's Impact to Date
$39.2M
Direct Investment in Fragile X Research
43
Teams Actively Researching Fragile X Syndrome
34
Pharmaceutical and Biomedical Partners
703
Fragile X Research Grants Awarded