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.
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.
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.
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.
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.
Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option
Harmony Biosciences’ RECONNECT Phase 3 trial of ZYN002 offered an at-home participation option. Unfortunately the trial reported negative results.
Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments
FRAXA-DVI is revolutionizing Fragile X syndrome research, providing efficient, comprehensive and objective preclinical testing of potential treatments.
Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice
Treating Fragile X might require a combination of drugs. FRAXA-DVI tested two investigational drugs in Fragile X mice. Together ibudilast and gaboxadol rescued a wide array of symptoms.
Unveiling Probiotic Potential in Fragile X Syndrome Clinical Trial
First of its kind in Serbia, this clinical trial explores probiotic intervention as a potential treatment avenue for Fragile X syndrome.
Pharmacological Modulation of Nicotinic Signaling
This study tests whether blocking certain nicotine-sensitive receptors in the brain during adolescence can improve attention and cognition in Fragile X.
Astrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome
This team studied how faulty calcium signaling in astrocytes contributes to sensory hypersensitivity in Fragile X, aiming to find new astrocyte-targeted treatments.
SRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome
This $100,000 FRAXA grant will fuel the Smith lab’s new approach to treating Fragile X syndrome using Saracatinib, originally a cancer drug.
Coming Together for Rare Disease Day 2023
Today we mark Rare Disease Day. FRAXA is committed to advancing research on Fragile X, one of the most common rare diseases worldwide.
Human FMR1 Isoform-Specific Regulation of Translation and Behavior
FMRP has multiple forms, and this team will study which isoforms are most important for brain development. This is key for future FMRP replacement therapies.
Recruiting: Clinical Study of Non-Invasive EEG for Children Ages 2-7
Dr. Carol Wilkinson, MD PhD at Boston Children’s Hospital is recruiting children ages 2-7 years with Fragile X syndrome to participate in a study of EEG.
Correcting Fragile X Syndrome Deficits by Targeting Neonatal PKCε Signaling in the Brain
Enhancing PKCε in early development normalized oxytocin, AMPAR signaling, and adult behavior in Fragile X mice, highlighting PKCε as a promising therapeutic target.
Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome
FRAXA funded the LovaMiX trial of lovastatin + minocycline for Fragile X. 2022 results show safety and support continued study of this dual-target treatment approach.
Purposeful and FRAXA Partnership Leads to Clinical Trial
AI and FRAXA-DVI identified a drug + supplement combo that reversed all Fragile X symptoms in mice. A clinical trial tested this in adults with Fragile X.
Contribution of Microglia to the Therapeutic Effects of Metformin and Adiponectin in Fragile X Syndrome
Why are some with Fragile X always hungry or overweight, yet rarely diabetic? This team is studying metabolism and testing treatments like metformin and diet.
Link Between Lipid Profile, eCBome System and Gut Microbiome in Fragile X Syndrome
Why does obesity challenge so many people with Fragile X? Dr. Caku’s team has found that Fragile X syndrome causes changes in the tiny organisms that live in our gut.
Preclinical Testing of High Fat/Low Carb Diets in Fragile X Mice and Cells
Dr. Cara Westmark’s team will use mice to determine if palatable Atkins-type diets can improve sleep and boost learning skills for those with Fragile X syndrome.
Pharmacotherapeutic Effects of Cannabidiol (CBD) in Fragile X syndrome (FXS) and Autism Spectrum disorder (ASD)
This study tested CBD (cannabidiol) treatment in male and female Fragile X mice to learn how and why it works and whether gender affects responses to CDB treatment.