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FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

Accelerating Fragile X Research for Effective Treatments

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Author: FRAXA Research Foundation

To Interrogate the Developmental Timing for Treating Fragile X Syndrome

FRAXA Research Foundation July 29, 2024November 19, 2024

Are there critical periods in Fragile X syndrome? Will treatment work in adults as well as in children? This team aims to answer these questions.

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QurAlis and UMass Chan Advance Fragile X Syndrome Treatment using ASOs (Antisense Oligonucleotides)

FRAXA Research Foundation June 25, 2024June 25, 2024

Explore how QurAlis and UMass Chan are revolutionizing Fragile X syndrome treatment using advanced ASO technology, setting new standards in therapeutic development.

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Exploring Advances Fragile X Research: Comprehensive Webinar Highlights – May 2024

FRAXA Research Foundation June 22, 2024May 14, 2025

Discover the latest in Fragile X research with insights from our May 2024 webinar. Learn about new FRAXA grants, key clinical trials, and scientific updates that are shaping the future of Fragile X syndrome treatments.

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Celebrate World Fragile X Day: July 22, 2024 – Global Landmarks Light Up

FRAXA Research Foundation May 29, 2024May 29, 2024

Celebrate World Fragile X Day on July 22, 2024 by lighting up landmarks. Join us in honoring Fragile X research and supporting affected families worldwide.

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Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option

FRAXA Research Foundation May 20, 2024May 19, 2025

Harmony Biosciences is now recruiting for the RECONNECT Phase 3 clinical trial of ZYN002, a potential treatment for Fragile X syndrome. The trial offers an at-home participation option, making it accessible for patients across the US, Australia, and the UK. Join us in this groundbreaking study and contribute to the future of Fragile X syndrome treatment.

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FRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research

FRAXA Research Foundation May 3, 2024May 12, 2025

Help accelerate research on Fragile X syndrome biomarkers by contributing samples to the COMBINEDBrain Consortium’s project. Contact Katie Clapp at FRAXA Research Foundation to learn how you can participate.

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Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments

FRAXA Research Foundation March 13, 2024

Explore how the FRAXA Drug Validation Initiative is revolutionizing Fragile X syndrome treatment, leading the charge towards innovative therapies and hope for affected families.

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Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome

FRAXA Research Foundation March 7, 2024March 3, 2025

Join Dr. Tsai and Dr. Kumar on a journey into novel treatment avenues for Fragile X syndrome. Learn how activating mGluR7 could be a game-changer, opening up uncharted therapeutic territory.

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Celebrating Teamwork: FRAXA’s 7th Year at Bruins 50/50 Event

FRAXA Research Foundation February 7, 2024March 11, 2024

Celebrating FRAXA’s 7th year at Bruins 50/50 Event: teamwork and community unite for Fragile X research.

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Shape Our Conversation: FRAXA Feedback Survey

FRAXA Research Foundation February 1, 2024February 2, 2024

Join FRAXA’s mission in shaping a brighter future for Fragile X research. Share your thoughts in our brief survey by February 15 and help us tailor our communication to your interests. Your insights are invaluable in our 30th year of progress.

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Recruiting: Unveiling Probiotic Potential in Fragile X Syndrome Clinical Trial

FRAXA Research Foundation December 20, 2023November 19, 2024

First of its kind in Serbia, this clinical trial explores probiotic intervention as a potential treatment avenue for Fragile X syndrome.

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$100,000 Matching Challenge From The Robert & Ardis James Foundation

FRAXA Research Foundation December 8, 2023December 8, 2023

We are thrilled to announce FRAXA Research Foundation’s most significant and unique matching challenge of the year, thanks to the Robert & Ardis James Foundation. This challenge will help us bring top new talent to Fragile X research.

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Pharmacological Modulation of Nicotinic Signaling

FRAXA Research Foundation December 6, 2023November 19, 2024

Nicotine — familiar to any smoker — tickles nicotinic acetylcholine receptors in the brain. These receptors are key to important brain functions including learning and memory. This team will explore whether drugs that dampen these receptors can improve cognitive function in Fragile X.

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Astrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome

FRAXA Research Foundation December 6, 2023November 19, 2024

Most Fragile X research has focused on one type of brain cells: neurons. But mounting evidence point to problems with astrocytes, star-shaped cells which are vitally important to normal brain function. This team is working to understand how astrocytes are involved in Fragile X and develop treatment approaches that targets astrocytes alone.

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Your Support on Giving Tuesday 2023 is Transforming Research

FRAXA Research Foundation November 29, 2023

On Giving Tuesday 2023, the generosity of 191 donors raised $333,517 for FRAXA Research Foundation, advancing research and hope for a cure for Fragile X syndrome.

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An Update from Harmony Biosciences on Giving Tuesday

FRAXA Research Foundation November 28, 2023November 28, 2023

Harmony Biosciences recently completed its acquisition of Zynerba Pharmaceuticals. I am excited for us to progress their work in pursuit of a treatment option for people living with Fragile X syndrome.

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Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

FRAXA Research Foundation November 7, 2023November 19, 2024

FRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.

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Virtual Research Q&A with Mike Tranfaglia, MD, and Katie Clapp

FRAXA Research Foundation November 2, 2023November 2, 2023

Please join FRAXA co-founders, Katie Clapp and Dr. Michael Tranfaglia, for a Research Q&A via Zoom on Wednesday, November 15, 2023 at 12:00 pm (noon) ET

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C-subunit Mitochondrial Leak Channel in Fragile X Syndrome

FRAXA Research Foundation September 20, 2023November 19, 2024

Explore Yale’s groundbreaking study on mitochondrial leak channels, set to revolutionize Fragile X syndrome treatment. Funded by a $100,000 FRAXA grant.

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Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome

FRAXA Research Foundation September 7, 2023November 19, 2024

Awarded a FRAXA Research grant, Dr. Andrew Stanfield, Dr. Leena E. Williams, and Dr. Damien Wright are set to explore somatosensory processing (sense of touch) in Fragile X syndrome at the University of Edinburgh. Their aim? A noninvasive touch test that could set the stage for future clinical trials in FXS.

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Antisense Oligonucleotides (ASOs) to restore FMRP in Human Fragile X Cerebral Organoids

FRAXA Research Foundation August 24, 2023November 19, 2024

Explore Dr. Richter’s encouraging results with ASOs for Fragile X syndrome. A $100,000 grant now fuels pivotal studies needed to advance toward ASO therapy.

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FRAXA Research Foundation Partners with Autism BrainNet

FRAXA Research Foundation August 17, 2023August 30, 2023

Discover how FRAXA Research Foundation’s collaboration with Autism BrainNet accelerates Fragile X syndrome research by collecting vital postmortem brain tissue. Dive into the significance of brain studies for deeper insights and potential therapeutic interventions.

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ASOs and Fragile X: Addressing the Most Asked Questions

FRAXA Research Foundation August 16, 2023

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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Support the FRAXA Dailey Family Campaign

FRAXA Research Foundation August 15, 2023August 15, 2023

Join the Dailey Family in their journey with Fragile X syndrome. Meet Kristin, a renowned marathon runner, Sean, with strong Boston roots, and their four children, including Declan with Fragile X.

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Slack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice

FRAXA Research Foundation August 14, 2023November 19, 2024

Learn how a $100,000 FRAXA research grant supports Yale researchers in using Slack potassium channel inhibitors to treat Fragile X syndrome by normalizing behaviors in FMR1 knockout mice.

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