Finding Fragile X Biomarkers – From Transcriptomics to Behavior in Patients

FRAXA funded a study using blood-based transcriptomics to find reliable Fragile X biomarkers. This unique approach links molecular data to behavior for future trials.

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Meng Li, PhD, Xinyu Zhao, PhD, and Anita Bhattacharyya, PhD

FRAXA Research Grants Drive Big Investments in Fragile X

FRAXA is working with hundreds of university labs and more than 30 pharmaceutical companies around the world. Dr. Michael Tranfaglia spends a lot of his time advising and collaborating with industry partners.

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Healx team 2018

Repurposing Study II: Evaluating Combinations of Drugs to Treat Fragile X

FRAXA partnered with Healx to use AI to find approved drugs and drug combos that could treat Fragile X. Top candidates are now being tested in Fragile X models.

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Healx team David, Dan, Narissa - FRAXA (1)

Drug Repurposing Study Results Accelerate Progress Towards Fragile X Treatments

Drug repurposing leverages the detailed information available on approved drugs and reduces the time and money needed to deliver safe “new” treatments, with greater success rates and quicker impact.

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Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives

FRAXA is 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.

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Identifying Biomarkers for Fragile X Syndrome – A Study in Argentina

FRAXA funds biomarker research to develop objective, biological measures of Fragile X, helping predict treatment response, guide trials, and move therapies toward FDA approval.

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New compound from Anavex Improves Learning and Behavior in Fragile X Mice

The investigational drug Anavex 2-73 was able to improve intellectual, learning and hyperactivity measures in a mouse model of Fragile X syndrome. This is a sigma-1 receptor agonist.

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bugula

Bryostatin Restores Learning and Memory in Adult Fragile X Mice

A marine critter found off the California coast — Bugula neritina — is the only source of Bryostatin. Bryostatin-1 restores spatial learning and memory in adult Fragile X mice.

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FRAXA Funded Research

Current Research Grants (37)