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!
Researchers at the University of Alabama are looking for members of the FX community to take their online survey so they can explore the knowledge & opinions of the FXS community on pharmacogenomic testing.
Dr. Elizabeth Berry-Kravis and Dr. Walter Kaufmann joined us for a 45-minute webinar where they presented on two of the "top 10 most-cited articles" in the American Journal of Medical Genetics Part A. Presentations were then followed by a moderated Q&A.
As gene therapies continue to enter rare disease and Fragile X spaces, we know that our community has become increasingly curious about what this means for Fragile X. At the 19th NFXF International Fragile X Conference, experts shared their thoughts on gene therapies and their future in the Fragile X world.
Researchers at Purdue University are conducting a natural history research study to learn about brain activity in females, ages18-60 years, living with the FMR1 premutation.
Three of our industry partners working on treatments for Fragile X syndrome (FXS) presented updates on their research at the 19th NFXF International Fragile X Conference on July 27, 2024. Presenters included representatives from Tetra Therapeutics, Harmony Biosciences, and Spinogenix.
Congratulations to our 2024 Junior Investigators! We are SO excited to have such brilliant minds in the Fragile X field. The NFXF is proud to support these Junior Investigators and build the next generation of Fragile X experts.
David Hessl, PhD, joined us for a 45-minute webinar where he presented the results of a longitudinal study focusing on executive function changes in people with the FMR1 premutation. The presentation was followed by a moderated Q&A.
