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