Dr. Thomas Maurin and Dr. Barbara Bardoni, Fragile X researchers and co-authors of a 2025 review on PDE inhibitors published in Cell Reports Medicine.

Fragile X and PDE Inhibitors: A Promising Path Forward for Brain Disorders

Fragile X syndrome research shows PDE inhibitors may improve brain function and behavior, with promise for related neurodevelopmental disorders.

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Urgent Action Needed: Help Secure NIH Funding for Fragile X Research

NIH funding delays threaten Fragile X research, putting critical studies and future treatments at risk. Help us urge Congress and the NIH to act now.

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Advancing Curative Therapy for Fragile X Syndrome: Turner Lab Secures $1M Grant

Fragile X syndrome research accelerates as the Turner Lab secures $1M to expand FRAXA-funded work on curative therapy targeting the missing FMRP protein.

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FRAXA-Funded Research Explores ISRIB as a Potential Treatment for Fragile X

ISRIB for Fragile X syndrome is being studied as a potential treatment to restore brain function and social behavior. Researchers investigate its effects.

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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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Callum Cup VIII Scores $19,400 for Fragile X Research – A Milestone Event

Callum Cup VIII, Millburn FC’s annual charity match, scored big with $19,400 raised for Fragile X research, bringing total funds to $148,000!

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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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19th Annual Fragile X Poker Run Raises $33,000 for Fragile X Research!

The 19th Annual Fragile X Poker Run, hosted by Jimmy, Dana & Mikey Charlton, brought friends & family together in Sparta, NC.

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Kicking Off Our 2024 Giving Season With Gratitude and Hope

FRAXA’s 2024 Annual Appeal begins with a $100K gift from the Berg and Schatz families, funding new Fragile X research teams and advancing a cure.

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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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World Fragile X Day 2024: A Global Celebration of Awareness and Research Progress

Recap of World Fragile X Day 2024, a global event spearheaded by FRAXA. Explore beautiful photos of landmark illuminations around the world.

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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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Hole-in-One for Fragile X Research: Highlights from the 37th Annual Charity Golf Tournament

The Hall family’s golf tournament raised $35,000 for Fragile X research—bringing their total contributions to over $327,000 for FRAXA!

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Patrick’s PALS 28th Annual Basketball Tournament for FRAXA Breaks Record

Recap of Patrick’s PALS 28: a successful 3-on-3 basketball fundraiser in Boston, raising $170,000+ for FRAXA Research Foundation.

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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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Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration

FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.

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FRAXA Funded Research

Current Research Grants (40)