Stephanie Barnes, PhD

Enhancing NMDA Receptor Signaling to Treat Fragile X Syndrome

FRAXA-backed work revealed NMDA receptors may hold the key to correcting brain signaling in Fragile X, pointing to new treatment strategies.

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High-resolution image of dendritic spines—tiny structures on neurons that are altered in Fragile X syndrome.

New Research Targets NMDA Receptor – A Key Player in Brain Communication and Fragile X Syndrome

Fragile X syndrome research identifies NMDA receptor as a promising treatment target, with drugs already in trials offering faster paths to human studies.

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Mark Bear, PhD & Sara Kornfeld Simpson

Altered Physiology of Primary Visual Cortex in Fragile X Syndrome

This team believes inhibitory neurons expressing somatostatin are impaired in Fragile X. They will see if stimulating these neurons has therapeutic potential.

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Allos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen

Discover Allos Pharma’s advancements in a pivotal Phase 3 trial for Fragile X syndrome treatment, Arbaclofen. Learn how their FDA-informed trial design might finally bring hope to the Fragile X community.

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20 Years of Advancing Fragile X Research: Progress Toward a Cure

MIT Professor Mark Bear traces the discoveries that give us great optimism of finding effective treatments and ultimately a cure for Fragile X syndrome.

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Drug Tolerance in MGluR5 Clinical Trials – Dr Patrick McCamphill 1:1 with FRAXA

We suspected that clinical trials of mGluR5 blockers from Novartis and Roche failed because the drug triggered tolerance. Dr. Patrick does find tolerance, and is looking for ways to overcome it.

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Developing Arbaclofen for Fragile X – Dr. Mark Bear 1:1 with FRAXA

MIT professor Dr. Mark Bear founded Allos Pharma to develop arbaclofen (STX209) as a treatment for Fragile X syndrome. Dr. Bear sat down with FRAXA co-founder Katie Clapp to share the story and next steps.

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Allos Pharma Revives Arbaclofen After 7 Years: New Hope for Fragile X Syndrome

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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Bear lab (Bear 3rd from left, McCamphill on right)

Scientists Find a New Way to Reverse Symptoms of Fragile X

MIT Professor Mark Bear and colleagues have identified a new target for Fragile X therapeutics: GSK3 alpha. Several FRAXA research teams previously identified GSK3 beta as a treatment target.

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Newly discovered aspects of fragile X spur next wave of drugs

Spectrum News – Newly Discovered Aspects of Fragile X Spur Next Wave of Drugs

Many drugs for Fragile X syndrome have failed in large clinical trials, but candidates that target new aspects of the condition may fare better.

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Patrick McCamphill

Pharmacological Tolerance in the Treatment of Fragile X Syndrome

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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Mechanisms of Tolerance to Chronic mGluR5 Inhibition

FRAXA supported research showing mGluR5 antagonist tolerance develops quickly in Fragile X models, guiding new strategies to prevent or overcome it.

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Development of a High-Content Synapse Assay to Screen Therapeutics for Fragile X Syndrome

This work established a high-content synaptic imaging platform for Fragile X cells to test many candidate drugs for their ability to repair synapse structure and function.

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Mark Bear lab - Fragile X

Mark Bear’s Goal: Disease-Modifying Treatments for Fragile X

Mark Bear pioneered the mGluR theory of Fragile X, linking excess protein synthesis to symptoms and driving development of disease-modifying treatments now tested in clinical trials.

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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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Role of Excessive Protein Synthesis in the Ontogeny of FXS

Excessive neuronal protein synthesis is not just a symptom but appears to cause early synaptic wiring defects in Fragile X — highlighting translation control as a key target.

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FRAXA Funded Research

Current Research Grants (37)