This project explores the role of oligodendrocytes in Fragile X. The team will test if improving these support cells’ function can restore normal brain activity to treat Fragile X.
Read moreAuthor: FRAXA Research Foundation
FRAXA Drug Validation Initiative (FRAXA-DVI)
The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS).
Read moreShionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
Shionogi’s EXPERIENCE clinical trials for Fragile X syndrome are nearing completion and enrollment for the adult and adolescent trials are closed. Learn more about the study, FRAXA’s role, and the open-label extension.
Read moreSex Differences and the Role of Estrogen Receptors in Fragile X
Fragile X syndrome researchers are studying how estrogen receptors shape brain activity and may explain why males and females experience symptoms differently.
Read moreFragile X Syndrome Research in 2025: FRAXA Announces New Grants & Promising Leads
Fragile X syndrome research is advancing in 2025 with new FRAXA-funded grants supporting breakthrough science, therapy platforms, and clinical tools.
Read moreRole of Microglia in Fragile X Syndrome
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.
Read moreModeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids
Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA.
Read moreUMass Chan Medical School Licenses RNA-Based Therapy to QurAlis, Advancing a New Era in Fragile X Treatment
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.
Read moreHarvard’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.
Read more#teamwally: Help Find a Cure for Fragile X Syndrome
Join #teamwally in supporting Fragile X research. Read Wally’s inspiring journey and help fund a future of hope and breakthroughs for kids like him.
Read moreNew 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.
Read moreFragile 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.
Read moreFragile 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.
Read moreUrgent 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.
Read moreAdvancing 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.
Read moreHelp 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!
Read moreGEXVal 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.
Read moreHelp 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.
Read moreHonor 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.
Read moreSupport 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.
Read moreDonate 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!
Read moreSupport 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.
Read moreBK 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.
Read moreInvestigating 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.
Read moreNPR 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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