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.

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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.

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Frank Kooy lab

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.

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Galarneau lab team

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.

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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.

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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.

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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.

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Patrick McCamphill

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.

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Craig Erickson lab

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.

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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.

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Tori Shaeffer

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.

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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.

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Taurine and Somatostatin as Potential Treatments for Fragile X Syndrome: A Unifying Neuro-Endocrine Hypothesis

March 21, 2007

Dr. Abdeslem El Idrissi’s FRAXA-funded study tested somatostatin and taurine in Fragile X mice, revealing taurine’s promise as an accessible therapy.

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FRAXA Funded Research

Current Research Grants (37)