The gene that, when mutated, causes Fragile X syndrome.
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- Coming Full Circle – Kathy May Returns back to FRAXACo-Founder Kathy May returns after two decades to share her story. It’s about discovering new forms of treatments to enhance the mental, emotional and social growth of those affected by Fragile X. “And there will be a cure,” she said. “FRAXA is the reason for this hope. I have come full circle to FRAXA and feeling more hopeful than I have in many years.”
- Reactivating the FMR1 Gene to Reverse Fragile X SyndromeFRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.
- Positive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor Zatolmilast from Tetra TherapeuticsToday, Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome, press release below. The results do not depend on carving out a subset of patients or post hoc analysis.
- Contribution of Microglia to the Therapeutic Effects of Metformin and Adiponectin in Fragile X SyndromeThe research team of Brian Christie, PhD and Marie-Eve Tremblay is developing ways to balance hormones, including drugs like metformin and changes in diet, which could not only reduce hunger and obesity, but ultimately also improve learning and behavior in Fragile X syndrome.
- Recruiting: Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home OptionHarmony Biosciences is now recruiting for the RECONNECT Phase 3 clinical trial of ZYN002, a potential treatment for Fragile X syndrome. The trial offers an at-home participation option, making it accessible for patients across the US, Australia, and the UK. Join us in this groundbreaking study and contribute to the future of Fragile X syndrome treatment.