Fragile X Research Update: A Turning Point for Treatments and Curative Approaches

Fragile X research is at a turning point. FRAXA is funding ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.

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Evaluating Novel Drug Candidates for Fragile X Using the Live Mouse Tracker

This grant is funding AI-driven drug discovery, advanced mouse behavior tracking, and gene expression analysis to uncover new treatments for Fragile X syndrome.

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Versatile Drug Screening Platform for Fragile X Syndrome

Combinations of drugs may be needed to treat Fragile X syndrome. How can we find the best combinations in the ideal doses? This project tackles this challenge.

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Frank Kooy lab

GABA-A Receptor in Fragile X Syndrome

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

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Ganaxolone Fragile X Clinical Trial Showed Disappointing Results

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

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

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

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

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Frank Kooy, PhD, at University of Antwerp

Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome

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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Frank Kooy lab

A Kinase Assay as a Biomarker for Fragile X Syndrome

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

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FRAXA Funded Research

Current Research Grants (38)