The NFXF Blog
Kaerus Bioscience Completes Successful Phase 1 Trial, Demonstrating Promise for KER-0193 BK Channel Modulator for Fragile X Syndrome
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!
An Xtraordinarily Successful Third – and Last – Fragile X Christmas Party!
The Haugen’s third Fragile X Christmas Party was another smashing success. Their fundraising goal was $25,000, and they raised a whopping $36,420.
Welcome Class of 2025 Board Members
Executive Director Hilary Rosselot welcomes the incoming class of 2025 Board Members. We look forward to working together to serve the entire Fragile X community.
Single-Dose Study for Adult Men with FXS
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.
2025 NIH Fragile X Centers of Excellence — Webinar
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.
ACTION ALERT:
Contact Congress About the Importance of NIH Research Funding for Fragile X
We are seeing numerous proposed changes this Congress that would have a significant impact on the Fragile X community. It’s more important than ever that your members of Congress hear directly from you — [...]
ACTION ALERT:
Advocate for Medicaid and the Fragile X Community
Action Alert- Advocate for Medicaid protection and the Fragile X community.
Gene therapies in action: a look at examples of successful treatments
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!
EXPERIENCE Clinical Trials – Join Today!
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.
NFXF Webinar Series: Gene Therapy & the FXS Community: A Review of Community Surveys
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.
NFXF Belonging Project: Site Updates
Check out the latest updates from the Belonging Project research sites! The Belonging Project, one of our newest initiatives, aims to intentionally extend our reach to underserved and underrepresented communities across the United States. We have updates from three Fragile X clinics and our belongingness survey, and will keep you updated as we continue to move forward.
Preparing for the Return to Routine After a Long Break: Tips for Educators, Employers, and Caregivers
The return after a long break can be a difficult transition for anyone, especially an individual living with FXS. With thoughtful planning and a compassionate approach, the entire support team can help ease the process.