Celebrating 20 Years of the Fragile X Poker Run in Sparta, NC
The 20th Annual Fragile X Poker Run in Sparta, NC celebrated two decades of community, music, and fundraising for Fragile X research.
Shionogi Shares Update on Zatolmilast Fragile X Clinical Trials
Shionogi shares an update on its zatolmilast Fragile X clinical trials, outlining progress in U.S. studies and next steps in data analysis and evaluation.
Gene Therapeutic Development for Fragile X Syndrome
Dr. Lee’s team is testing RNA editing gene therapy for Fragile X, aiming to repair FMR1 RNA and restore missing protein — targeted, reversible, promising.
FMRP Regulatory Role in Human Hippocampal Development and Therapeutic Interventions in Fragile X
Fragile X syndrome hippocampal organoids show neuron–glia imbalance. This team will map disrupted gene networks and test PDE inhibitors to restore brain function.
Fragile X Research Q&A: Dr. Berry-Kravis on the RECONNECT Trial
Fragile X Research Q&A with Dr. Berry-Kravis on RECONNECT results, placebo effects, outcome measures, methylation, and research directions for families.
RECONNECT Trial Results: ZYN002 Does Not Meet Primary Endpoint in Fragile X
Harmony Biosciences announced that the Phase 3 RECONNECT trial of ZYN002 in Fragile X syndrome failed to meet its primary endpoint.
Validate a Novel Antisense Oligonucleotide Therapeutic for Fragile X Syndrome
FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder.
Fragile X Research Update: A Turning Point for Treatments and Curative Approaches
Fragile X research is at a turning point. FRAXA is funding ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.
ASO Rescue of FMR1 Mis-Splicing in Neurons and Mitigation of Fragile X Deficits
A new FRAXA grant funds UMass Chan researchers using ASOs in neurons and organoids to correct FMR1 mis-splicing and restore critical FMRP protein.
BK Channel Openers: From FRAXA Seed Funding to Big Pharma Investment and Trials
See how FRAXA’s early grants propelled Fragile X research from lab discoveries to industry momentum, with partners advancing therapies into trials.
Evaluating Novel Drug Candidates for Fragile X Using the Live Mouse Tracker
This grant is funding AI-driven drug discovery, advanced mouse behavior tracking, and gene expression analysis to uncover new treatments for Fragile X syndrome.
Metabolic and Mitochondrial Crosstalk in Insulin and cAMP Pathways in Fragile X Syndrome
Fragile X syndrome research explores insulin and cAMP pathway crosstalk, mapping brain metabolism to guide treatment strategies.
Oligodendrocytes: a Potential Route to Treat Fragile X Syndrome
This project explores the role of oligodendrocytes in Fragile X. The team will test if improving these cells’ function can restore normal brain activity to treat Fragile X.
FRAXA Drug Validation Initiative (FRAXA-DVI)
The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing to validate investigational and repurposed compounds for Fragile X.
Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
Learn more about Shionogi’s EXPERIENCE clinical trials for adults and adolescents with Fragile X syndrome, FRAXA’s role, and the open-label extension of these trials.
Sex 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.
Modeling 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.
Fragile 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.
A Tat-Conjugate Approach to Treat Fragile X Syndrome
Turner Lab’s innovative approach to treating Fragile X uses a Tat-linked, truncated FMRP protein designed to restore brain function by replacing the missing protein.
Role 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.
UMass 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 gene-targeted therapy closer to clinical trials.
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.
#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.
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.