Bridging Naturalistic Behavior and Nucleus Accumbens in Fragile X Syndrome
How do sex and gene dosage interact to shape brain function and behavior in Fragile X syndrome? The team aims to uncover why symptoms differ between males and females.
Recruiting: Mirum Launches Clinical Trial of MRM-3379 for Fragile X Syndrome
Mirum’s MRM-3379 Phase 2 trial builds on FRAXA-funded PDE4D research and is now enrolling males ages 13–45 with Fragile X syndrome. View eligibility and study sites.
Targeting Serotonin 1a Receptors to Reverse Neurobehavioral Phenotypes
Neurolixis’ new drug targets serotonin 1A receptors (aka 5-HT1A), showing promise in preclinical studies for Fragile X syndrome, funded by a FRAXA grant for future clinical trials.
Spinogenix and FRAXA Advance SPG601 Into Phase 2b Trial for Fragile X Syndrome
Spinogenix and FRAXA are supporting a Phase 2b clinical trial of SPG601 in Fragile X syndrome, building on Phase 2 signals and FDA designations.
GEXVal Completes FRAXA-DVI, Receives FDA Orphan Drug Designation, and Prepares for Phase 2a Trial
Orphan Drug Designation positions GXV-001 for next steps after FRAXA-DVI completion as GEXVal prepares a Phase 2a Fragile X trial in Europe.
Harnessing FMRP Autoregulation for Safer Gene Therapy in Fragile X
Fragile X syndrome gene therapy could be safer with an FMRP “safety dial” that self-regulates expression using natural FMR1 control elements.
Nospharma Announces Partnership with FRAXA to Test NOS-01 in Pre-Clinical Models of Fragile X
NOS-01 enters the FRAXA Drug Validation Initiative (FRAXA-DVI) for Fragile X mouse-model testing, generating data to guide development decisions.
Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier
A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.
Pharmacological Activation of PGC-1α to Treat Fragile X Syndrome
Dr. Tsai and Dr. Acharya are testing whether boosting mitochondrial function can improve key Fragile X syndrome features in a mouse model and inform treatment strategies.
FRAXA Drug Validation Initiative (FRAXA-DVI)
The FRAXA Drug Validation Initiative (FRAXA-DVI) provides streamlined, cost-effective preclinical testing to evaluate investigational and repurposed compounds for Fragile X syndrome.
Help Rowan Thrive: Support Fragile X Research
Join the Gale and Kellogg families in supporting Fragile X research through FRAXA. Help Rowan and others thrive by funding life-changing treatments and advancing curative therapies.
Advanced Preclinical Testing of NKCC1 Inhibitors Supporting Phase 2 Trials in Fragile X
The project evaluates a next-generation NKCC1 inhibitor, studying its safety and effects on brain signaling to determine its potential to progress into Phase 2 clinical trials.
Exploring the Auditory System Therapeutic Target for Fragile X Syndrome
Auditory system therapeutic target for Fragile X syndrome research uses a rat model to connect excess protein synthesis with disrupted circuits and auditory hypersensitivity.
Donate to the Clark Family Campaign
Join Lucas 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!
Identifying New Therapeutic Approaches Using a Human Fragile X Model
Human Fragile X syndrome model research identifies new therapeutic approaches by revealing how FMRP loss affects human neurons and brain circuits.
Cell Type-Specific Protein Dysregulation in Fragile X Syndrome Brain Organoids
Study finds that brain cell types respond differently to gene silencing, pointing to the need for customized treatment approaches in Fragile X.
Pokémon Tournament Twitch Stream Raises $2,100 for FRAXA
Twitch streamer Zae hosted a Pokémon charity tournament that raised $2,100 for FRAXA and Fragile X research, uniting a global community through gaming for a cause.
METTL3 Inhibitors and FDA-Approved Drugs in a New Fragile X Treatment Strategy Using Organoids
Researchers are testing METTL3 inhibitors and FDA-approved drugs in brain organoids to explore new pathways for treating Fragile X and related disorders.
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 novel gene editing therapies for Fragile X, aiming to repair FMR1 and restore the missing protein — a promising new strategy which is targeted and reversible.
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.
Roles of Postnatal Transient Connectivity in the Development of Fragile X Syndrome
This team is studying why people with Fragile X are overly sensitive to sound and light, using advanced imaging to find brain changes and test ways to prevent them.
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.