Fragile X Research Q&A with Dr. Berry-Kravis on RECONNECT results, placebo effects, outcome measures, methylation, and research directions for families. The post Fragile X Research Q&A: Dr. Berry-Kravis on the RECONNECT Trial appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for 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. The post Oligodendrocytes: a Potential Route to Treat Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing to validate investigational and repurposed compounds for Fragile X. The post FRAXA Drug Validation Initiative (FRAXA-DVI) appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for 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. The post Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Fragile X syndrome researchers are studying how estrogen receptors shape brain activity and may explain why males and females experience symptoms differently. The post Sex Differences and the Role of Estrogen Receptors in Fragile X appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Fragile X syndrome research is advancing in 2025 with new FRAXA-funded grants supporting breakthrough science, therapy platforms, and clinical tools. The post Fragile X Syndrome Research in 2025: FRAXA Announces New Grants & Promising Leads appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for 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. The post Role of Microglia in Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA. The post Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
UMass Chan Medical School licenses RNA-based Fragile X treatment approach using ASOs to QurAlis, moving gene-targeted therapy closer to clinical trials. The post UMass Chan Medical School Licenses RNA-Based Therapy to QurAlis, Advancing a New Era in Fragile X Treatment appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Dr. Jeannie Lee wins $1M Blavatnik Award to advance FMR1 gene reactivation therapy for Fragile X, building on years of FRAXA-funded research. The post Harvard’s Dr. Jeannie Lee Wins $1M Award to Develop Gene Reactivation Therapy for Fragile X appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Harmony Biosciences announced that the Phase 3 RECONNECT trial of ZYN002 in Fragile X syndrome failed to meet its primary endpoint. The post RECONNECT Trial Results: ZYN002 Does Not Meet Primary Endpoint in Fragile X appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure 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. The post Validate a Novel Antisense Oligonucleotide Therapeutic for Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
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. The post Fragile X Research Update: A Turning Point for Treatments and Curative Approaches appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
A new FRAXA grant funds UMass Chan researchers using ASOs in neurons and organoids to correct FMR1 mis-splicing and restore critical FMRP protein. The post ASO Rescue of FMR1 Mis-Splicing in Neurons and Mitigation of Fragile X Deficits appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
See how FRAXA’s early grants propelled Fragile X research from lab discoveries to industry momentum, with partners advancing therapies into trials. The post BK Channel Openers: From FRAXA Seed Funding to Big Pharma Investment and Trials appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for 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. The post A Tat-Conjugate Approach to Treat Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Cognitive rigidity shapes daily life in Fragile X, driving anxiety and repetitive behaviors. Learn its impact on treatment and independence. The post Cognitive Rigidity: a Hallmark of Fragile X and Autism appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
This grant is funding AI-driven drug discovery, advanced mouse behavior tracking, and gene expression analysis to uncover new treatments for Fragile X syndrome. The post Evaluating Novel Drug Candidates for Fragile X Syndrome Using the Live Mouse Tracker appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Fragile X syndrome research explores insulin and cAMP pathway crosstalk, mapping brain metabolism to guide treatment strategies. The post Metabolic and Mitochondrial Crosstalk in Insulin and cAMP Pathways in Fragile X Syndrome appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
Fragile X syndrome fundraising took center stage at Patrick’s PALS 29, raising over $150,000 at Boston’s premier 3-on-3 basketball event. The post Patrick’s PALS 29 Raises Over $150K for Fragile X Syndrome at 3-on-3 Tournament appeared first on FRAXA Research Foundation - Finding a Cure for Fragile X Syndrome.| FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome