NFXF Blog

We explain where the challenges come from, what makes FXS treatment different from other conditions, and how to start building a clearer picture of your options.

We ask the Fragile X community to share any specialists who have helped them with FXPOI and who might be interested in learning more about the consortium.

Carme Torrents, MD, a 2026 Jr. Investigators Awardee, summarizes an important symposium by Drs. Elizabeth Smith, Tatyana Adayev, and Anne Wheeler, during the 20th International Fragile X Conference.

Watch the 2026 Summer Scholars research project presentations by Erin Burnett, Iris Chen, Nura Salem, and Tim Smith.

Tetsuya Asano, a 2026 Jr. Investigators Awardee, summarizes an important talk by Jessica Klusek during the 20th International Fragile X Conference.

A new study from the CDC and NFXF examines geographic access to Fragile X specialty care and identifies gaps in access to FXCRC clinics across the U.S.

New research uncovers how gene expression, protein regulation, and synaptic pathways are altered in Fragile X syndrome and FXTAS, offering deeper insight into disease biology.

Survey results reveal strong interest in gene therapy for Fragile X syndrome, highlighting family perspectives, hopes, concerns, and future research needs.

Learn more about Mirum Pharmaceuticals, a biopharmaceutical company conducting clinical trials to investigate potential treatments for rare diseases.