FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder.
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Fragile X Research Update: A Turning Point for Treatments and Curative Approaches
Fragile X research is at a turning point. Two late-stage clinical trials, zatolmilast (EXPERIENCE) and ZYN002 (RECONNECT), could soon deliver the first approved treatments. FRAXA is also funding innovative approaches like ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.
Read moreASO 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.
Read moreBK 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.
Read moreA Tat-Conjugate Approach to Treat Fragile X Syndrome
Explore Turner Lab’s novel approach to developing definitive treatment for Fragile X syndrome with a Tat-conjugated, truncated FMRP protein. This innovative strategy aims to restore brain circuit function and reduce abnormal behaviors linked to Fragile X by directly addressing the cause of Fragile X: a missing protein.
Read moreEvaluating Novel Drug Candidates for Fragile X Syndrome 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.
Read moreMetabolic 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.
Read moreOligodendrocytes: 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.
Read moreFRAXA 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 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 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 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 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 moreFRAXA-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.
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 moreAltered 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.
Read moreGene 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.
Read moreFragile 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.
Read moreMarvel 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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