FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder.
Read more2025 Grants
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.
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 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 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 moreTo Interrogate the Developmental Timing for Treating Fragile X Syndrome
Are there critical periods in Fragile X syndrome? Will treatment work in adults as well as in children? This team aims to answer these questions.
Read moreSomatosensory Processing as a Therapeutic Target for Fragile X Syndrome
FRAXA-funded researchers in Edinburgh assessed a noninvasive touch test that could be used for clinical trials in Fragile X syndrome.
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