Project Mercury - Three-year progress report on global FSHD trial readiness
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1.
FSHD Global Research Foundation
- 2. World FSHD Alliance, Germany
- 3. FSHD UK, United Kingdom
- 4. FSHD Canada Foundation, Canada
- 5. ABRAFEU, Brazil
- 6. FSHD Spain, Spain
- 7. FSHD Stichting, Netherlands
- 8. FSHD Society
- 9. FSHD Italia, Italy
- 10. FSHD Society, USA
Description
Facioscapulohumeral muscular dystrophy (FSHD) is a rare, inherited neuromuscular disease affecting as many as 1 in 8,000 individuals worldwide. With therapeutic programs advancing into Phase 3 clinical trials, the prospect of approved treatments has never been greater. Rare disease drug development faces systemic barriers: insufficient clinical trial sites, limited patient enrolment, lack of HTA-ready evidence, and inadequate healthcare system readiness. In a resource-constrained ecosystem, fragmented approaches risk slowing progress for all. To address this, the FSHD Society, in partnership with global FSHD patient advocacy organisations (PAOs), launched Project Mercury in 2023. Building on the success of Project Hercules in DMD, Project Mercury is a patient-led, global, multi-stakeholder initiative designed to advance FSHD clinical trial readiness and therapeutic access through coordinated, collaborative action.
Files
Project Mercury-Three-year progress report on global FSHD trial readiness and patient access.pdf
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