Building a Clinical Data Library

FOCUS will unify clinical trial, natural history, and patient data 

By Dr. Lucienne Ronco, FSHD Society 

---

As part of the Global FSHD Innovation Hub, the FSHD Society has undertaken the development of a clinical data library, the FSHD Open Collaborative Unified System (FOCUS).  FOCUS will house all forms of FSHD clinical data whether derived from natural history studies like ReSOLVE or MOVE, or from therapeutic clinical trials like those performed by Fulcrum Therapeutics.  

Reflecting the strong relationship built between the Society and Fulcrum, Fulcrum has offered to share all data from their Phase 2 and 3 clinical trials of losmapimod in FSHD.  Fulcrum has shared both treated and placebo (non-treated) data and also all of the patient and clinician reported and muscle function data that was collected during these studies. In addition, Fulcrum collected serum and plasma samples from each individual throughout the course of the trials. The Society is working with Fulcrum and Sanofi to determine if these samples can be made available for research. 

This exceptionally gracious gift is intended to serve as a foundation for FSHD clinical knowledge. It will facilitate current and future clinical trial designs to test novel therapeutics. This FSHD patient clinical data will also be used to better understand disease etiology, improve patient selection, optimize clinical trial design, inform endpoint selection, support market access submissions, and answer partner-specific data questions. 

---

This article was originally published in the The FSHD Advocate, 2025 Issue 2. Read the latest issue here.