GABA Archives

Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice

March 6, 2024

Treating Fragile X might require a combination of drugs. FRAXA-DVI tested ibudilast and gaboxadol in Fragile X mice. Together they rescued a wide array of symptoms.

Functional and Genomic Characterization of Interneurons in the Fmr1-KO Mouse Brain

September 22, 2022

The brain’s balance is maintained by two types of neurons: excitatory and inhibitory. This team has found fewer than normal inhibitory cells in Fragile X mice.

Repurposing FDA-Approved Drugs to Treat Major Depressive Disorder in Fragile X Syndrome

April 24, 2022

Depression is common in Fragile X, but current antidepressants need FMRP to work. Researchers will screen FDA-approved drugs to find effective options for FXS.

GABA-A Receptor in Fragile X Syndrome

November 24, 2021

Published results from Dr. Frank Kooy reveal how GABAA receptor changes may hold the key to treating Fragile X syndrome.

Clinical Trial of Metformin for Fragile X Syndrome

October 24, 2021

FRAXA-funded open-label trial found that metformin led to increased GABA-mediated cortical inhibition, suggesting metformin modulates core Fragile X pathways.

Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model

November 23, 2020

Long-term, but not short-term, treatment with bryostatin-1 arrested some behavioral and cognitive symptoms in a mouse model of Fragile X.

Allos Pharma Revives Arbaclofen After 7 Years: New Hope for Fragile X Syndrome

November 19, 2020

Allos Pharma Inc launches a new development program for arbaclofen to treat Fragile X syndrome. This experimental drug missed its primary endpoint in clinical trials.

Ganaxolone Fragile X Clinical Trial Showed Disappointing Results

February 7, 2019

Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.

Qionger He, PhD, and Anis Contractor, PhD

NKCC1 Inhibitor Bumetanide Corrects Synaptic and Circuit Hyperexcitability in Fragile X Mouse Model

November 15, 2018

Dr. Anis Contractor and Dr. Qionger He investigated the potential of the available drug bumetanide to correct altered GABA signalling in a mouse model of Fragile X syndrome.

Pharmacological Tolerance in the Treatment of Fragile X Syndrome

May 9, 2018

FRAXA funded MIT work to probe tolerance to key Fragile X drugs, including mGluR5 inhibitors and arbaclofen, and to identify ways to sustain long-term treatment benefits.

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.

18th International Fragile X and Related Neurodevelopmental Disorders Workshop, Quebec, Canada

In Their Own Words: Reports From the International Fragile X Workshop

October 29, 2017

The 18th International Fragile X Workshop in Quebec was a great success, featuring more Fragile X research than ever before!

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

October 25, 2017

Dr. Erickson’s trial targeted brain imbalance in Fragile X by boosting GABA signaling with AZD7325, aiming to restore excitatory–inhibitory balance and improve symptoms.

Frank Kooy, PhD, at University of Antwerp

Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome

September 2, 2017

Dr. Frank Kooy and colleagues conducted a double blind crossover trial of ganaxolone in patients with Fragile X with FRAXA funding. Results of this study were mixed.

A Kinase Assay as a Biomarker for Fragile X Syndrome

January 15, 2017

Dr. Frank Kooy at the University of Antwerp investigated whether phosphorylation abnormalities are a suitable biomarker for clinical trials in Fragile X syndrome.

The Endocannabinoid System in a Mouse Model of Fragile X Syndrome

March 4, 2015

Fragile X disrupts endocannabinoid signaling. This study in mice demonstrated that correcting it may calm brain hyperexcitability and improve symptoms.

Functional Interplay Between FMRP and CDK5 Signaling

September 13, 2014

FRAXA-funded work showed CDK5 signaling is disrupted in Fragile X. CDK5 drugs are in development for Alzheimer’s so this pathway offers a promising new FX treatment angle.

Development of a Novel GABA-A Agonist in Fragile X Syndrome

September 9, 2013

FRAXA funded analysis of a selective GABA-A drug for Fragile X, leading to a clinical trial at Cincinnati Children’s to test this potential treatment.

What Works, and What Doesn’t

September 7, 2012

Early on, no one knew which path would work. Now the results are clear, and they’re directing FRAXA toward the next major Fragile X treatment breakthrough.

A Metabolomic Drug Efficacy Index to Test Treatments in the Fragile X Mouse

September 1, 2012

This work revealed small-molecule metabolic changes in Fragile X brains and is using them to build a drug-efficacy index for screening therapies.

GABAergic Inhibitory Function in Fragile X Syndrome

February 1, 2011

Fragile X mice show weakened GABAergic inhibition in key brain regions like the amygdala. Enhancing GABA_A receptor activity reduced hyperactivity and improved inhibition.