NFXF Blog

Mirum Pharmaceuticals has officially enrolled the first participant in its BLOOM Phase 2 clinical study evaluating MRM-3379, a potential new treatment for Fragile X syndrome (FXS).

This research looks at how a child’s level of intellectual disability (ID) — meaning how much their thinking and learning abilities are affected — relates to the types of behavioral challenges they experience if they have Fragile X syndrome (FXS).

CONNECTA Therapeutics is taking a new approach to Fragile X syndrome treatment by boosting the brain’s natural ability to reconnect and adapt.

Harmony Biosciences recently announced results from its Phase 3 RECONNECT study of ZYN002, a cannabidiol (CBD) gel being tested for Fragile X syndrome.

Bowen’s FX Therapeutics, a company with roots in the Fragile X community, is advancing efforts toward a future protein replacement therapy.

The Belonging Project aims to intentionally extend our reach to underserved and underrepresented communities across the United States. We have 2025 updates from three Fragile X clinics and our belongingness survey, and we will keep you updated as we continue to move forward.

Announcing FY 2025 federal research funding opportunities across two award categories available for all Fragile X-associated conditions and disorders.

We’re on a mission to enroll 600 participants with FXS born in 2003 through 2020. We’re halfway there and we need your help!

FXTAS was first described in five grandfathers of children with Fragile X syndrome in 2001 by Dr. Randi Hagerman and her colleagues.