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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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Marine Anais Krzisch, PhD

Role of Microglia in Fragile X Syndrome

FRAXA Research Foundation June 26, 2025June 26, 2025

Learn how Dr. Marine Anais Krzisch’s $35K FRAXA and ASF-funded project uses human iPSC microglia models to uncover pathways for Fragile X syndrome treatment.

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Jeannie Lee, MD, PhD and Chloe Chen, PhD

Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids

FRAXA Research Foundation June 18, 2025

Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA.

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UMass Chan Medical School Licenses RNA-Based Therapy to QurAlis, Advancing a New Era in Fragile X Treatment

FRAXA Research Foundation June 5, 2025

UMass Chan Medical School licenses RNA-based Fragile X treatment approach using ASOs to QurAlis, moving a groundbreaking gene-targeted therapy closer to human clinical trials.

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Dr. Jeannie Lee standing in front of a staircase at Harvard Medical School, recipient of the Blavatnik $1M award for Fragile X gene reactivation research

Harvard’s Dr. Jeannie Lee Wins $1M Award to Develop Gene Reactivation Therapy for Fragile X

FRAXA Research Foundation May 30, 2025June 2, 2025

Dr. Jeannie Lee wins $1M Blavatnik Award to advance FMR1 gene reactivation therapy for Fragile X, building on years of FRAXA-funded research.

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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

FRAXA Research Foundation April 30, 2025

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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Together for a Brighter Future: Celebrating the 5th World Fragile X Day

Holly Roos April 17, 2025April 22, 2025

Celebrate the 5th World Fragile X Day on July 22! Join a global movement lighting up landmarks to raise awareness and support families affected by Fragile X.

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Researcher analyzing gait patterns on a treadmill for Fragile X syndrome motor function study.

Fragile X and Fatigue: Study Reveals How Muscle Function is Altered in Children

FRAXA Research Foundation April 16, 2025

FRAXA-funded research shows how Fragile X syndrome muscle function affects walking and fatigue, offering insights for future treatments.

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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

FRAXA Research Foundation March 26, 2025

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

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Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome

FRAXA Research Foundation March 21, 2025May 19, 2025

Shionogi’s EXPERIENCE clinical trials for Fragile X syndrome are nearing completion. Enrollment for the adult trial (EXPERIENCE-301) is now closed, while the adolescent trial (EXPERIENCE-204) is in its final phase. Learn more about the study, FRAXA’s role, and the open-label extension.

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

FRAXA Research Foundation March 13, 2025March 13, 2025

NIH funding delays are threatening Fragile X research, putting critical studies and future treatments at risk. FRAXA Research Foundation and NFXF are urging Congress and the NIH to act now. Learn how you can help secure funding for Fragile X research.

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

FRAXA Research Foundation March 10, 2025

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

Anna Devine February 26, 2025March 6, 2025

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

FRAXA Research Foundation February 17, 2025February 28, 2025

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

Andrew Murphy January 10, 2025January 10, 2025

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. Discover how soccer unites for a cause.

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

FRAXA Research Foundation January 2, 2025January 2, 2025

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!

Holly Roos November 22, 2024December 18, 2024

Friends, family, and supporters came together again at the Alleghany Inn in Sparta, NC, for the 19th Annual Fragile X Poker Run, continuing a powerful tradition started by organizers Jimmy and Dana Charlton.

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Kicking Off Our 2024 Giving Season With Gratitude and Hope

Katie Clapp November 14, 2024May 8, 2025

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

FRAXA Research Foundation October 22, 2024

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

FRAXA Research Foundation October 7, 2024June 26, 2025

FRAXA Research Foundation awards a $100,000 grant to Ludwig Maximilian University researchers to investigate the role of insulin-degrading enzyme (IDE) in Fragile X syndrome, exploring new therapeutic approaches for cognitive and metabolic challenges in FXS.

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NPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome

FRAXA Research Foundation October 1, 2024April 21, 2025

NPR spotlights zatolmilast, a promising drug offering new hope for individuals with Fragile X syndrome. Families report life-changing improvements in anxiety, communication, and independence.

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Mark Bear, PhD & Sara Kornfeld Simpson

Altered Physiology of Primary Visual Cortex in Fragile X Syndrome

FRAXA Research Foundation September 19, 2024May 30, 2025

Discover how a $100,000 FRAXA grant supports research at MIT targeting neuron dysfunction in Fragile X, aiming to develop new therapies to improve sensory processing and behavior.

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Gene Editing of FMR1 to Correct FXS Phenotypes in Mice

FRAXA Research Foundation September 5, 2024May 30, 2025

FRAXA awarded a $100,000 grant to Dr. Davidson and Dr. Yrigollen at Children’s Hospital of Philadelphia to explore gene editing tools aimed at treating Fragile X syndrome by correcting FMR1 mutations in a unique mouse model.

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Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier

FRAXA Research Foundation August 21, 2024November 19, 2024

Discover how a $100,000 FRAXA grant awarded to Cincinnati Children’s Hospital is advancing Fragile X research by simplifying 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

FRAXA Research Foundation August 20, 2024August 30, 2024

Discover how Marvel Biosciences and FRAXA Research Foundation are collaborating to test MB204, a promising new treatment for Fragile X syndrome, building on groundbreaking adenosine receptor research.

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

Holly Roos August 12, 2024

Recap of World Fragile X Day 2024, a global event by FRAXA that highlighted research advances and raised awareness for Fragile X syndrome. Explore beautiful photos of landmark illuminations around the world.

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