A Turning Point in Fragile X Research: Treatments and Curative Approaches скачать в хорошем качестве

A Turning Point in Fragile X Research: Treatments and Curative Approaches

2025 may be the year Fragile X research delivers what families have waited decades to see: the first approved treatments and bold steps toward a cure. Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and a leading single-gene cause of autism. For families, it brings daily challenges with learning, behavior, and independence. For researchers, it has meant decades of steady progress toward treatments that can make a real difference. This video highlights why 2025 may be a historic year for Fragile X research. Two late-stage clinical trials, zatolmilast (EXPERIENCE) and ZYN002 (RECONNECT), are nearing results. Either success could deliver the first approved treatment for Fragile X. FRAXA is also supporting therapies that address the root cause of Fragile X. Antisense oligonucleotide (ASO) therapy at UMass Chan aims to restore the missing Fragile X protein, while CRISPR-based gene reactivation at Harvard has shown near-complete success in patient-derived neurons. New testing models like organoids and the Live Mouse Tracker are helping speed the path from lab discovery to clinical impact. For the Fragile X community, this could be the year we move from decades of waiting into a new era of hope, real treatment options, and the path toward a cure. FRAXA’s mission is to find effective treatments and ultimately a cure for Fragile X syndrome. We directly fund research grants and fellowships at top universities around the world. We partner with biomedical and pharmaceutical companies, large and small, to bridge the gap between research discoveries and actual treatments. Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders. To learn more about FRAXA's Fragile X syndrome research, to get involved with Fragile X awareness and events or to donate to FRAXA research, visit www.fraxa.org Let's Connect Facebook:   / fraxaresearch   Instagram:   / fraxaresearch   LinkedIn:   / fraxa-research-foundation   Reddit:   / fragilexreseach   Website: https://www.fraxa.org