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
FRAXA-DVI Study Finds Promising Results for Marvel Biosciences’ A2A Receptor Drug in Fragile X Model
FRAXA-DVI study finds Marvel Biosciences’ MB-204, an A2A receptor blocker, improved behavior and cognition in a Fragile X mouse model.
Neurodevelopmental Splicing Defects in Fragile X Syndrome Brain Models
Neurodevelopmental splicing defects in Fragile X syndrome are studied with human brain organoids to identify RNA changes and potential ASO therapies.
Untangling the Mystery of Sensory Overload in Fragile X Syndrome
This FRAXA grant supports research on how altered brain signaling may contribute to sensory overload in Fragile X syndrome and related behaviors.
FRAXA's Impact to Date
$38.9M
Direct Investment in Fragile X Research
41
Teams Actively Researching Fragile X Syndrome
34
Pharmaceutical and Biomedical Partners
701
Fragile X Research Grants Awarded