FRAXA Research Foundation Partners with Autism BrainNet

FRAXA’s collaboration with Autism BrainNet can accelerate Fragile X syndrome research by collecting vital postmortem brain tissue.

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ASOs and Fragile X: Addressing the Most Asked Questions

Explore the potential of ASOs in treating Fragile X syndrome & FXTAS. Dive into a comprehensive Q&A addressing key questions and breakthrough findings.

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In Memory of Garret

Honor Garret Peacock Volker’s extraordinary life by supporting FRAXA’s mission to find a cure for Fragile X syndrome. Your donation can make a difference in the lives of those affected.

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Pioneering Community-Based Drug Development for Fragile X Syndrome

Discover how FRAXA leverages Community-Based Drug Development to create impactful therapies for Fragile X syndrome. Join us as we reshape the future of rare disease treatment.

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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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2023 Fragile X Research Banbury Meeting (1)

Breakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies

Explore the latest breakthroughs in Fragile X research unveiled at the recent Banbury Meeting. Discover novel strategies, from gene therapy to protein replacement, that bring hope for curative therapies.

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Coming Together for Rare Disease Day 2023

Today we mark Rare Disease Day. FRAXA is committed to advancing research on Fragile X, one of the most common rare diseases worldwide.

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Validating Novel Inhibitors of ERK Signalling to Treat Fragile X Syndrome

This team is testing ERK inhibitors — drugs that may calm overactive brain signaling in Fragile X. Early results in mice show strong benefits with minimal side effects.

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mRNA Therapy for Fragile X Syndrome

Dr. Kathryn Whitehead helped develop the science behind the COVID-19 vaccines. Her team adapted this technology to deliver the Fragile X mRNA to brain cells.

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A Note of Thanks and Hope on This Giving Tuesday

Fragile X research matters. Each discovery brings us closer to treatments for autism and other brain disorders. Thanks to you, we have hope.

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Repurposing FDA-Approved Drugs to Treat Major Depressive Disorder in Fragile X Syndrome

Depression is common in Fragile X, but current antidepressants need FMRP to work. Researchers will screen FDA-approved drugs to find effective options for FXS.

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VLM Commodities Charity Week Raises $10,000 for Fragile X Research

“Living with Fragile X is what we do—we keep going forward,” Lou shared. “FRAXA gives purpose, and I’m proud to support Charity Week.”

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Aggression, Other Fragile X Behaviors Tend to Ease Over Teenage Years

Behavioral problems such as hyperactivity and aggression are generally more frequent in younger children with Fragile X syndrome, becoming less common as they grow through adolescence and journey toward adulthood, researchers report.

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Gordon Research Conference for Fragile X and Autism-Related Disorders, Explained

The Fragile X and Autism-Related Disorders Gordon Research Conference is a biannual event that provides an international forum for the presentation and discussion of frontier research in these conditions. Dr. Mike Tranfaglia explains why this is the premier conference for researchers and the scientific community.

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Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome

FRAXA funded the LovaMiX trial of lovastatin + minocycline for Fragile X. 2022 results show safety and support continued study of this dual-target treatment approach.

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10 Year Vision for Collaborations That Transform Fragile X and Autism Research

The future offers hope for people living with Fragile X syndrome. Collaborations between the Fragile X community and other disability organizations help to provide understanding and advancement of research to bring effective treatments to families. FRAXA’s Dr. Mike Tranfaglia talks with Autism Science Foundation’s Allison Singer about the importance of their collaboration as we look forward to the next 10 years.

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Cannabinoids as a Treatment for Fragile X Syndrome

This team uses EEG to study sensory hypersensitivity in Fragile X. By testing drugs in mice, they aim to find treatments that calm brain overactivity.

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2021 Fragile X Research Grants Funded by FRAXA Research Foundation

Each year, FRAXA funds a diverse portfolio of research. Our FRAXA Fellowships are seed funding for the future, the feedstock for the Fragile X treatment development pathway. While we are looking to promote as many promising new approaches as possible, prominent themes emerge each year, as scientists around the world tackle previously neglected areas.

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Promising Results of Preclinical Study of ANAVEX®2-73

We are excited to share that Anavex Life Sciences announced today that preclinical data of the ANAVEX®2-73 (blarcamesine) study in Fragile X syndrome were published in the peer-reviewed journal, Scientific Reports.

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Fragile X Clinical Trials

Synaptogenix Announced Intention to Launch a Fragile X Clinical Trial with Bryostatin

Bryostatin research has advanced from mouse models to human trials. Synaptogenix and Nemours make plans to test this potential treatment in Fragile X clinical trials.

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Fragile X Syndrome: In Pursuit of a Cure Webinar

Global webinar “Fragile X Syndrome: In Pursuit of a Cure” on July 22, 2021 commemorated World Fragile X Day. Over 5,000 registered from more than 50 countries.

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Pharmacotherapeutic Effects of Cannabidiol (CBD) in Fragile X syndrome (FXS) and Autism Spectrum disorder (ASD)

This study tested CBD (cannabidiol) treatment in male and female Fragile X mice to learn how and why it works and whether gender affects responses to CDB treatment.

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Elizabeth MCullough and Achim Klug

Auditory Dysfunction in Fragile X Syndrome in a Mouse Model of Fragile X

FRAXA-funded studies found Fragile X mice show altered auditory circuit function with delayed startle timing and reduced prepulse inhibition, mirroring human sound sensitivity.

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Towards Understanding the Role of FMRP in Human Brain Development Using Brain Organoids

Dr. Zhexing Wen and Dr. Peng Jin of the newly funded Fragile X Center of Excellence at Emory University School of Medicine join us in this seminar to present about Understanding the Role of FMRP in Human Brain Development Using Brain Organoids.

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FRAXA Funded Research

Current Research Grants (38)