Help Direct Millions in Fragile X Research Funding – DOD Seeks Reviewers

The DOD’s Fragile X syndrome research program seeks reviewers to help evaluate funding applications. No science background needed—apply by March 7!

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GEXVal and FRAXA Collaborate to Advance Fragile X Research with Phase 2a Trial

GEXVal and FRAXA collaborate to advance Fragile X research with the Phase 2a trial of GXV-001, supported by AMED’s funding program.

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Rowan, a young girl with Fragile X syndrome, standing with her family at a scenic outdoor location, symbolizing love and support.

Help Rowan Thrive: Support Fragile X Research

Join the Gale family in supporting Fragile X research through FRAXA. Help Rowan and others thrive by funding life-changing treatments and advancing curative therapies.

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Discovering Effective Treatments & A Cure for Fragile X Syndrome

Honor Cooper’s Birthday with a Donation to Fragile X Research

Honor Cooper’s birthday by donating to Fragile X syndrome research. Every contribution supports new treatments and hope for a brighter future.

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Support the FRAXA Schutz Family Campaign & #teamwally Campaign

Join the Schutz family in their journey with Fragile X syndrome. Discover Wally’s inspiring progress, the impact of clinical research, and how donations to FRAXA fuel hope for breakthroughs to support families affected by Fragile X.

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Donate to the Clark Family Campaign

Join Stefanie Clark’s heartfelt appeal to support Fragile X research. Help fund breakthroughs for Lucas and others like him. Every dollar makes a difference — donate today!

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Support the Stevenson Family Campaign

The march of time renews the commitment we made to a special needs community 25 years ago. We vowed to dream big and never give up until there were effective treatments available and eventually a cure for Fragile X syndrome, the most commonly inherited cause of intellectual disabilities and autism.

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BK Channel Openers: A New Hope for Fragile X Treatment – Insights from Kaerus Bioscience CEO Robert Ring

Kaerus Bioscience’s BK channel openers for Fragile X syndrome are advancing through Phase 1 trials, offering hope for new treatments with FRAXA’s continued support.

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Investigating the Role of the Insulin Degrading Enzyme (IDE) in Fragile X Syndrome

This study explores how disrupted insulin signaling affects metabolism and brain function in Fragile X, revealing new treatment targets for both body and mind.

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NPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome

NPR spotlights zatolmilast, a promising investigational drug to treat Fragile X syndrome. Families report life-changing improvements in learning and independence.

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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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Gene Editing of FMR1 to Correct FXS Phenotypes in Mice

This team is testing cutting-edge gene editing tools to correct Fragile X mutations in the brain. Using a new mouse model, they will see if treatment can restore normal function.

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Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier

A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.

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Marvel Biosciences Partners with FRAXA to Test MB204 for Fragile X Syndrome

Marvel Biosciences and FRAXA Research Foundation are collaborating to test MB204, a promising new treatment for Fragile X which targets adenosine receptors.

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Identification of the Proteome of Active and Silenced FMR1 Alleles in Human Stem Cells

This project aims to uncover which proteins keep the Fragile X gene silenced. By identifying them, the team hopes to find new ways to switch the FMR1 gene back on.

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Understanding Anxiety in Fragile X: Insights from Dr. Carrie Buchanan’s Webinar

Dr. Carrie Buchanan shares strategies to manage anxiety in individuals with Fragile X syndrome, including non-pharmacological treatments and medication options.

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To Interrogate the Developmental Timing for Treating Fragile X Syndrome

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)

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

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

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

Harmony Biosciences sponsored the RECONNECT Phase 3 clinical trial of ZYN002 to treat Fragile X syndrome. The trial offered an at-home participation option, making it more accessible for patients.

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FRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research

Help accelerate research on Fragile X syndrome biomarkers by contributing samples to the COMBINEDBrain Consortium’s project.

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Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments

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

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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FRAXA Funded Research

Current Research Grants (46)