Fragile X Advocacy Newsletter – April 2025
Read and share the latest edition of the Fragile X Advocacy Newsletter with your Members of Congress offices!
New Resources– Genetics of Fragile X
The Genetics of Fragile X can be confusing. We’ve developed a series of handouts to help explain how changes in the FMR1 gene happen, how Fragile X is inherited, and what CGG repeat ranges mean for individuals and families.
Help Us Reach the FORWARD-MARCH Enrollment Goal
We’re on a mission to enroll 600 participants with FXS born in 2003 through 2017. We’re halfway there and need your superhero help to hit our target!
Genetics of Fragile X — Webinar
Susan Howell, Rena Pressman, Marisa Vomvos, and Kate Shelly joined us for a one-hour Q & A discussing the Genetics of Fragile X.
ACTION ALERT:
Protect the Department of Education
Action Alert- Protect the Department of Education to ensure continued protections for all students, especially members of the Fragile X community.
Advocate for Fragile X From Home
Advocating from home is a key part of ensuring continued support for individuals with Fragile X. Let’s work together to make our voices heard!
Potential Impact of the Continuing Resolution on CDMRP/PRMRP Funding Opportunties
The Congressionally Directed Medical Research Program could face a 57% reduction in funding with the proposed continuing resolution.
ACTION ALERT:
Contact Congress Securing NIH funding for Fragile X
We continue to see proposed changes this Congress that may have a significant impact on the Fragile X community. It’s more important than ever that your members of Congress hear directly from you — [...]
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!
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.
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 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.