NFXF Original Research Articles
The original research articles here were written by Fragile X professionals within the FXCRC and NFXF team to address critical needs in Fragile X research and treatment.
The authors discuss a variety of recommendations made with the vision that effective new treatment will lie at the intersection of innovation, rigorous and reproducible research, and stakeholder involvement.
Results from a survey to investigate the main symptoms, daily living challenges, family impact, and treatment priorities for individuals with FXS and their families. It was completed by 467 participants, including 439 family members or caretakers of someone with FXS, 20 professionals who work with a person with FXS, and 8 individuals with FXS.
Caregiver reports, clinical observations, and diagnostic assessments indicate that most individuals with Fragile X syndrome experience high levels of chronic anxiety. However, anxiety is a challenging endpoint for outcome measurement in FXS because most individuals cannot reliably report internal emotional or body states.
The final report and information on the Fragile X patient experience from our FDA NFXF-led patient-focused drug development meeting held on March 3, 2021.
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These are open-access articles distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. This encourages collaboration, and they are commonly referenced by industry and other professionals working on Fragile X.
The NFXF is dedicated to our vision of a world where every family is empowered to successfully navigate the Fragile X journey. To address the needs of the Fragile X community, we are proud to collaborate with Fragile X professionals who share our vision.
Thank you to the professionals who dedicate their careers to learning more about Fragile X. And thank you to the families who participated in the research summarized in these articles. We couldn’t do it without you!
Researchers interested in accessing this data, please visit the NFXF Data Repository.