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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Harvard’s Dr. Jeannie Lee Wins $1M Award to Develop Gene Reactivation Therapy for Fragile X
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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New Research Targets NMDA Receptor – A Key Player in Brain Communication and Fragile X Syndrome
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
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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Fragile X and Fatigue: Study Reveals How Muscle Function is Altered in Children
FRAXA-funded research shows how Fragile X syndrome muscle function affects walking and fatigue, offering insights for future treatments.
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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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Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
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
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
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. Discover how soccer unites for a cause.
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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!
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
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
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
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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Altered Physiology of Primary Visual Cortex in Fragile X Syndrome
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 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
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
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
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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Identification of the Proteome of Active and Silenced FMR1 Alleles in Human Stem Cells
This team’s ultimate goal is to find a way to restore full function of FMR1, the gene which underlies Fragile X syndrome. With this grant, they will use advanced technologies to find the specific proteins that are involved in keeping FMR1 silenced. Understanding precisely why and how the gene is silenced is a key step toward finding a solution.
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Understanding Anxiety in Fragile X: Insights from Dr. Carrie Buchanan’s Webinar
Discover effective strategies for managing anxiety in individuals with Fragile X syndrome. Learn from Dr. Carrie Buchanan’s insights on non-pharmacological treatments, common anxiety triggers, and current medication options to improve quality of life.
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