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.
Here's a message from our friends at Harmony Biosciences in advance of the 19th NFXF International Fragile X Conference.
In June 2024, NIH published RFA-HD-25-002: Centers for Collaborative Research in Fragile X and FMR1-Associated Conditions. If you or someone you know are researching FXS and/or FMR1-associated conditions, this could be a great funding opportunity for you!
Help the NFXF - share your thoughts on gene therapy.
Authors: David Hessl, PhD, Karina Mandujano Rojas, BS, Emilio Ferrer, PhD, Glenda Espinal, BS, Jessica Famula, MS, Andrea Schneider, PhD, Randi Hagerman, MD, Flora Tassone, PhD, and Susan M. Rivera, PhD Summary: People with [...]
The University of Kansas BRAIN Lab is conducting a research study to learn about behavioral and brain differences associated with the Fragile X premutation. Males and females ages 50-80 living with the Fragile X premutation, with or without FXTAS, may be eligible to participate. The study includes remote & in-person visits at the University of Kansas.
Researchers at the University of Memphis and Cincinnati Children's Hospital Medical Center are conducting a research study exploring the relationship between barriers and the FXS screening process among family members.
Researchers at the University of Maryland are looking to understand how children with Fragile X syndrome (FXS) and other intellectual disabilities grieve the loss of a loved one. If you are the parent or guardian of a child with FXS or another intellectual disability who has experienced the death of someone in their life when they were 5-17 years old, researchers want to talk to you.
The University of Washington's Neurodevelopmental Language and Learning (NeuDLL) Lab is conducting research addressing how children learn words in stories and the skills that support learning. Participation is remote and can be completed from home. Sessions are scheduled at your convenience.
Dr. Craig Erickson joined us for a 45-minute webinar where he presented the results of a single-dose medication study in FXS and then answered questions during a moderated Q&A.
