Yesterday, Kaerus Bioscience announced that it has successfully completed its Phase 1 trial and demonstrated 'proof of mechanism' with their novel BK channel modulator, KER-0193, being developed for Fragile X Syndrome!
Cincinnati Children's Hospital Medical Center is looking for males ages 18-40 with FXS to participate in a single-dose clinical trial that is studying a drug called Gaboxadol.
Hear updates about the NIH-funded Fragile X Centers of Excellence, which support research to improve the diagnosis and treatment of Fragile X syndrome and its related conditions.
Although there are currently no options for approved or experimental gene therapies for FXS, gene therapies in other diseases and conditions do exist and are helping people today! Read the blog to see some examples of gene therapy in the real world!
You may have heard about EXPERIENCE (Evaluation of Fragile X Experience in Cognition Expression) clinical trials as the Tetra studies or the studies of BPN14770 in Fragile X syndrome. EXPERIENCE is now being managed by Shionogi and clinical trial sites across the U.S. are still enrolling qualified male participants aged 9-45.
In December 2024, the NFXF put on a webinar discussing gene therapy and the perspectives from those in the FX community. During this webinar, expert Dr. David Hampson laid the groundwork of understanding what gene therapy is, followed by three different groups - The Patrick Wild Centre, Mt. Siani Hospital, and the NFXF - presenting on their community surveys, which all aimed to understand the perspectives on gene therapy from the Fragile X community.
The Belonging Project, one of our newest initiatives, aims to intentionally extend our reach to underserved and underrepresented communities across the United States. Hear from each of the three Fragile X clinics and our own in-house belonging survey, on how we’ve begun work to understand the challenges to diagnosis, treatment, and feeling a sense of community belonging faced by Black, Hispanic, and Native American groups and the providers who serve them.
Gene therapies are being developed at a rapid rate. Although there are currently no FDA-approved or interventional gene therapies for FXS, the future has never looked brighter for treating the root cause of FXS!
Researchers at the New York State Institute for Basic Research are conducting a study to better understand the development of physical (somatic) symptoms in females living with and without an FMR1 gene variation.
You Spoke, We Listened: The Completely At Home RECONNECT Clinical Trial –Participate in Research Without the Stress of Traveling
Researchers at Rush University Medical Center are working on technology to improve how to identify and track progress in children living with autism and Fragile X syndrome. The study is currently recruiting children ages 18 months to 5 years, and 12-18 years.
We live in a day and age of social sharing, and that is not going anywhere. However, when we participate in a clinical trial, we have to understand that we cannot share everything in order to protect the integrity of the trial.
Researchers at the Autism Assessment, Research, Treatment & Services (AARTS) Center at Rush University Medical Center are currently conducting a fully-virtual research study that is testing two telehealth interventions that are designed to help parents of children with genetic syndromes and intellectual disabilities gain strategies to manage challenging behaviors.
Movement disorders researchers at Rush University Medical Center are conducting a research study to learn about brain activation in people living with FXTAS during tasks like walking and thinking. This study is currently recruiting adults ages 50+ who are living with FXTAS.
We asked our 2024 NFXF-funded Randi J Hagerman Summer Scholars to summarize their summer project in a 15-minute video presentation, and here they are!
