IGF-1

Fragile X Treatment Strategy Emerges from FRAXA Research: IGF-1

Neuren’s Trofinetide, part of a promising IGF-1 drug class, showed standout results in Fragile X mice—outperforming Rett syndrome models.

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Dr. Emily Osterweil

Lovastatin Discovery in Fragile X Mice Leads FRAXA to Fund Clinical Trials

FRAXA honored Dr. Emily Osterweil for discovering that lovastatin can correct key Fragile X abnormalities. Her findings were published in Neuron.

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Phase 2b Clinical Trial of Arbaclofen in Autism Has Disappointing Results

The Phase 2b arbaclofen trial in autism didn’t improve social withdrawal, but did show progress on the Clinical Global Impression of Severity scale.

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Clinical Trials FAQ ← Frequently Asked Questions

Wondering which Fragile X trial is right? Eligibility varies, so most families qualify for just one. Talk with your closest clinic to find the best fit.

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FRAXA Progress and Future Plans

Huge momentum in Fragile X research: major results released, widespread media attention, and Phase III trials moving toward possible treatments.

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What Works, and What Doesn’t

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.

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Compound that Inhibits mGluR5 Corrects Signs of Fragile X in Adult Mice

A Roche and MIT study published in Neuron finds that an mGlu5 inhibitor, CTEP, can reverse many Fragile X symptoms in adult mice.

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FRAXA Announces 2012 Fragile X Research Awards

$1.13 million in FRAXA funding in 2012 supported new Fragile X research projects and renewals, advancing work toward better treatments.

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Fragile X Research Grants and Fellowships Funded 2011

Over $1 million in FRAXA funding in 2011 supported leading Fragile X research projects focused on developing effective treatments and future cures.

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FRAXA 2010 Research Awards Funded

Over $1.5 million in FRAXA funding supported Fragile X research and fellowships in 2010, empowering scientists to pursue breakthrough treatments.

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Results of First Fenobam Trial in Adults with Fragile X Published

Fenobam showed positive results in a Phase IIa Fragile X trial. FRAXA supplied data that helped the drug earn Orphan status and move into clinical testing.

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3 Researchers Honored at FRAXA Investigators Meeting

FRAXA’s 2008 Investigators Meeting brought together 150+ researchers from around the world to collaborate and speed new Fragile X therapies.

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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 (40)