Galarneau lab team

Clinical Trial of Metformin for Fragile X Syndrome

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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Interrogate the Functions of FMRP in Brain Development Using Stem Cells

Dr. Xinyu Zhao of the Waisman Center and Department of Neuroscience at University of Wisconsin-Madison joins us in this seminar to present Interrogate the Functions of FMRP in Brain Development Using Stem Cells.

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Mechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse

We hear from Devin K. Binder, MD, PhD, Professor, University of California at Riverside Medical School and Khaleel Razak, PhD, Professor, University of California at Riverside.

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Devon Binder, PhD; Iryna Ethell, PhD, Patricia Pirbhoy, PhD, at UC Riverside School of Medicine

Understanding and Reversing Hypersensitivity to Sounds in Fragile X Syndrome

This FRAXA grant studied why people with Fragile X are overly sensitive to sound and tested drug strategies to calm the brain’s overactive auditory circuits.

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Nahum Sonenberg

Effects of Metformin in Fmr1 Knockout Mouse Model of Fragile X Syndrome

Metformin, a safe diabetes drug, activates AMPK to rebalance protein synthesis. FRAXA-funded work investigated its potential to treat Fragile X.

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Funding opportunities - FRAXA investigators

Combinatorial Drug Treatment in a Model of Fragile X Syndrome using Novel Biomarkers

University of California researchers Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, explored drug combinations to limit hypersensitivity to sounds in Fragile X mice.  

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Nahum Sonenberg, PhD, 2017 Fragile X Research Grant

Metformin, Diabetes Drug, Potential Fragile X Treatment

Dr. Nahum Sonenberg’s research showed the diabetes drug metformin can correct key signaling defects in Fragile X, leading to clinical trials of this safe, repurposed therapy.

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Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives

FRAXA has focused on identifying existing, approved drugs that could be repurposed for Fragile X, allowing potential treatments to move faster and at lower risk than starting from scratch. We’ve worked to advance the most promising candidates toward real-world use.

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Iryna Ethell UC Riverside, FRAXA researcher

Fragile X Syndrome Treatment Target: MMP-9

This paper shows that MMP-9 dysregulation is a critical part of the pathology of Fragile X, and MMP-9 should be considered a major treatment target for Fragile X syndrome.

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Kendal Broadie

Matrix Metalloproteinase Therapeutic Treatments for Fragile X Syndrome

Dr. Broadie showed that MMP enzymes disrupt synapse development in Fragile X. MMP inhibitors (e.g. minocycline) improved connectivity and behavior in fruit flies.

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Effects of minocycline on vocal production and auditory processing in a mouse model of Fragile X

With FRAXA funding, Dr. Khaleel Razak and Dr. Iryna Ethell explored robust biomarkers relevant to the FXS and the efficacy of minocycline treatment.

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Encouraging Results from First Trial of Minocycline in Fragile X

A clinical trial of minocycline in children with Fragile X found significantly better global improvement vs. placebo, supporting its safety and potential.

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Iryna Ethell, PhD, at University of California

Role of Matrix Metalloproteinases (MMP-9) in Fragile X

With a $220,000 FRAXA grant, Dr. Iryna Ethell’s team at UC Riverside uncovered MMP-9’s role in Fragile X—leading to a major treatment strategy using minocycline.

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Iryna Ethell, PhD, at University of California

Researchers Propose Minocycline to Treat Fragile X

Researchers found that minocycline, an available drug that blocks MMP-9, may improve Fragile X symptoms. This discovery inspired clinical trials.

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FRAXA Funded Research

Current Research Grants (37)