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.
Opening the Gate: BK Channel Modulation as a Promising Treatment Strategy for Fragile X Syndrome
Fragile X syndrome research is opening the door to BK channel therapies that may calm hyperexcitable brain circuits and improve key symptoms.
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.
FRAXA Backs Upcoming Phase 2B Trial of SPG601 for Fragile X Syndrome
FRAXA Research Foundation is supporting a Phase 2b clinical trial of SPG601, an experimental therapy targeting BK channels in fragile X syndrome, following promising early results.
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.
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 speedy, cost-effective, objective preclinical testing to validate investigational and repurposed compounds for Fragile X.
Uncovering the Dynamics of FMR1 Using Spatial Transcriptomics for Fragile X
Scientists investigate how changes in RNA localization affect cognition and behavior in Fragile X, building knowledge toward more precise therapies.
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.
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.
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.
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.
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.
Cognitive Rigidity: a Hallmark of Fragile X and Autism
Cognitive rigidity shapes daily life in Fragile X, driving anxiety and repetitive behaviors. Learn its impact on treatment and independence.
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.
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.
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.