2024 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.
Summary of the publication, Insight and Recommendations for Fragile X Premutation Associated Conditions from the 5th International Conference on FMR1 Premutation
This summary gives a broad overview of the main themes discussed at the 5th International Conference on FMR1 Premutation in early 2023.
Dr. Liz Berry-Kravis Explains Important Updates about the Tetra Trials
Learn more from Dr. Liz Berry-Kravis about the important updates to the Tetra trials to make participation easier!
Survey: What do you think about at-home research visits?
Dr. Schmitt and the Cincinnati Fragile X Center are conducting a survey to learn about potential barriers and advantages of conducting research visits in the home.
Fragile X Clinical & Research Consortium and Outcome Measures Meeting
The FXCRC met in Denver, CO to discuss the status of treatment in Fragile X syndrome. A half-day outcome measure meeting was held with representatives from the FDA, industry, the FXCRC and families living with FXS.
FORWARD-MARCH 2023 — Webinar
Drs. Elizabeth Berry-Kravis and Nicole Tartaglia presented updates about FORWARD-MARCH, followed by a Question and Answer session covering the specifics of the study.
Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study
The National Fragile X Foundation is proud to be part of the Everylife Foundation’s newest report, The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study.
The 2023 RJH Summer Scholar Presentations Are Now Live!
We asked our 2023 NFXF-funded Randi J Hagerman Summer Scholars to summarize their summer project in a 15-minute video presentation, and here they are!
Study: Single Dose Study for Adult Women and Men with FXS
Dr. Craig Erickson at Cincinnati Children’s Hospital Medical Center is conducting a clinical trial to learn about if medication can change the brain’s response to sound, which may be abnormal in FXS.
DOD grant awarded for a FXTAS trial
PureTech Health has been awarded a DOD grant of up to $11.4 million from the DOD for their trial of LYT-300, oral formulation of allopregnanolone, in people with Fragile X-associated tremor/ataxia syndrome (FXTAS).
A Reflection on the CEO Commission’s 2023 Hill Day
NFXF Board Member Jed Seifert recently represented the NFXF at the CEO Commission's Hill Day. Jed shares his reflections on this powerful experience.
We’re Urging Lawmakers to Support the Accelerating Kids’ Access to Care Act
The NFXF is one of 215 organizations urging lawmakers to support the Accelerating Kids’ Access to Care Act. Every child deserves the best care—regardless of who they are, where they live, or their family’s income.
Antisense Oligonucleotide Rescue of CGG Expansion–Dependent FMR1 Mis-Splicing in Fragile X Syndrome Restores FMRP
One of the most exciting advancements being done in Fragile XS research today is antisense oligonucleotide (ASO) therapy.
RECONNECT Trial Announces New AT-HOME Option for Participation
RECONNECT Trial Announces New AT-HOME/VIRTUAL Option for Participation
Project WellCAST: Caregivers of Children with Fragile X Needed for a Research Study
The Kelleher Lab at Purdue University is conducting an NIH-funded clinical trial called Project WellCAST. Project WellCAST aims to understand how to best support caregivers of children with rare neurogenetic conditions, including Fragile X syndrome.