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
Shionogi Shares Update on Zatolmilast Fragile X Clinical Trials
Shionogi shares an update on its zatolmilast Fragile X clinical trials, outlining progress in U.S. studies and next steps in data analysis and evaluation.
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Gene Therapeutic Development for Fragile X Syndrome
Dr. Lee’s team is testing RNA editing gene therapy for Fragile X, aiming to repair FMR1 RNA and restore missing protein — targeted, reversible, promising.
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FMRP Regulatory Role in Human Hippocampal Development and Therapeutic Interventions in Fragile X
Fragile X syndrome hippocampal organoids show neuron–glia imbalance. This team will map disrupted gene networks and test PDE inhibitors to restore brain function.
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Patrick’s PALS 30th Annual 3-on-3 Basketball Tournament & Community Celebration
the TRACK at new balance, 91 Guest St, Boston, MA 02135
World Fragile X Day
FRAXA's Impact to Date
$37.7M
Direct Investment in Fragile X Research
46
Teams Actively Researching Fragile X Syndrome
33
Pharmaceutical and Biomedical Partners
655
Fragile X Research Grants Awarded