Ziyuan Guo, PhD, and Lu Lu, PhD, standing together in a research lab with laboratory equipment in the background.

METTL3 Inhibitors and FDA-Approved Drugs in a New Fragile X Treatment Strategy Using Organoids

November 4, 2025

Researchers are testing METTL3 inhibitors and FDA-approved drugs in brain organoids to explore new pathways for treating Fragile X and related disorders.

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Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier

August 21, 2024

A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.

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fNIRS to Measure Treatment Response in Young Children with Fragile X

February 13, 2020

The team tested functional near-infrared spectroscopy (fNIRS). fNIRS uses light sources and sensors on the scalp to build a heat map of the brain in action.

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Gene Therapy Translational Studies for Fragile X Syndrome

June 6, 2019

With FRAXA funding, researchers tested AAV gene therapy to restore FMRP in Fragile X mice, measuring safety, effectiveness, and brain activity to inform future trials.

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FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says

February 4, 2019

Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms what so many parents, caretakers, and educators suspect: people with fragile X want to be social, and it is anxiety – not lack of interest – which usually hold them back. If anxiety could be reduced, more sociability would likely follow. Dr. Erickson is a Fragile X expert and FRAXA investigator who is currently conducting a Fragile X clinical trial of an investigational new drug.

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Craig Erickson, MD, Cincinnati Children's Hospital

Fragile X Clinical Trial of AZD7325 in Adults

February 1, 2018

FRAXA funded a trial of AZD7325, a drug that boosts GABA(A), in adults with Fragile X. Led by Dr. Craig Erickson, it also tested innovative biomarkers for future trials.

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Craig Erickson lab

Brain Imbalance Target of Dr. Erickson’s New Clinical Trial

October 25, 2017

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.

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FRAXA Funded Research

Current Research Grants (39)