According to Dr. Erickson, AZD7325 is a drug that selectively boosts GABA neurotransmission in the brain. GABA is the primary neurochemical in the brain that blocks brain activation. GABA activity is in balance in the brain with Glutamate activity, which is the primary neurochemical that causes brain activation. In Fragile X, GABA activity is insufficient and glutamate activity is excessive, likely causing brain activity to be out of balance. AZD7325 attempts to correct parts of this imbalance by boosting the insufficient GABA activity in the brains of people with Fragile X.
Read moreSearch Results
Preclinical Testing of Sleep-Wake Patterns as an Outcome Measure for Fragile X
FRAXA Research Foundation awarded $122,000 over 2016-2018 to Dr. Cara Westmark at the University of Wisconsin at Madison for studies of sleep disorders in Fragile X syndrome.
Read moreIntegrating Human and Mouse Studies in Fragile X Syndrome – an NIH Center Approach
Presentations by:
Craig Erickson – Translational medicine and mechanistic studies of brain neurophysiology in Fragile X Syndrome: A NIH Center Overview
Ernest Pedapati – Network Mechanisms, Biomarkers, and Pharmacology of Fragile X Syndrome in Humans
Devin Binder – Network Mechanisms of Neurophysiology and Behavior in mouse models of Fragile X Syndrome
Kimberly Huber – FMRP Regulation of local and long-range neocortical circuits in the mouse: Links with EEG phenotypes
How Close are We to a Cure for Fragile X?
What will a cure for Fragile X look like? It’s a question we all ask at some point. Will it be a pill taken every day for life? Will it be combinations of medicines, tailored to each individual? Will it be a protein replacement injection once a week? Will it be a one-time gene therapy infusion that fixes every cell in the body at the same instant?
Read moreResources for Researchers
FRAXA’s primary mission is to speed up progress towards effective treatments and ultimately a cure for Fragile X syndrome. As “bottlenecks” are identified we will try to facilitate solutions and add resources for university and pharma researchers worldwide, so please contact us for additional scientific resource requests.
Read moreFragile X Research: 3 Areas of Focus
FRAXA Research Foundation is and always has been singularly focused on research to find effective treatments and ultimately a cure for Fragile X syndrome. Everything we do each day is centered on this mission. Our goal is to get treatments to families that will improve the lives of those we love affected by Fragile X. We want to share our current research priorities and explain why we continue to need your support.
Read moreFRAXA Drug Validation Initiative (FRAXA-DVI)
The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS).
Read moreShould You Participate in a Fragile X Clinical Trial?
A Fragile X clinical trial of a new PDE4D allosteric inhibitor from Tetra Therapeutics is nearly complete. Right now there are 3 remaining spots open to males 18-45 years of age with Fragile X syndrome. Dr. Elizabeth Berry-Kravis at the Rush University Medical Center in Chicago is leading this trial. The drug being studied has a unique mechanism of action that might improve cognitive and memory function.
Read moreBrain 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.
Read moreFRAXA’s Monthly Giving Program, the Chain of Champions
As a link in the Chain of Champions, your monthly gift will deliver lasting impact to families affected by Fragile X syndrome. 100% of your donation will go towards finding effective treatments and ultimately a cure as well as sharing discoveries with the Fragile X community so that all can benefit as quickly as possible.
Read moreHealx Drug Repurposing Programme for Fragile X Syndrome
David Brown, MD, PhD, Ivan Angulo-Herrera, PhD and Anthony Hall of Healx present about the Drug Repurposing Programme for Fragile X syndrome.
Read moreKetogenic Diet Eases Symptoms in Fragile X Male Mice
The Westmark laboratory continues to study sleep and rest-activity cycles in Fragile X mice as a potential outcome measure that correlates between preclinical and clinical research. The analysis of sleep EEG in the mice has proven more labor intensive than they anticipated, but the team is collaborating with Dr. Rama Maganti’s laboratory at UW-Madison on the development of computer scrips to speed up the analysis.
Read moreFRAXA Shop – Fragile X Merchandise
FRAXA Research Foundation shop – Fragile X merchandise including FRAXA swag and handcrafted items made by Fragile X families.
Read moreClinical Trials in Fragile X Syndrome
Explore the journey through Fragile X syndrome clinical trials, guided by the heartfelt perspectives of mothers Holly Roos and Katie Clapp. Understand how participation, which is free and adjustable, advances research and paves the way for FDA-approved treatments.
Read moreNews & Updates
Fragile X news and updates about Fragile X research advances, studies, clinical trials, grants and more.
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