Facioscapulohumeral muscular dystrophy (FSHD) is at a turning point, with multiple disease-modifying therapies in development. To ensure global access, clinical trial readiness across diverse regions is critical. This publication idea was initiated through Project Mercury in collaboration with others to offer a practical toolkit for new trial sites. Drawing on lessons learned from experienced centers, it outlines essential requirements for site eligibility, including personnel, facilities, and patient access, and highlights the role of international networks and registries. The paper introduces a standardized trial roadmap and provides strategies for collaboration with sponsors, patient engagement, and regulatory navigation. Importantly, this resource serves as a future-ready checklist for sites preparing to join FSHD trials, supporting efficient start-up and execution. By sharing best practices, the toolkit aims to expand global trial capacity and accelerate delivery of therapies for FSHD and other rare neuromuscular disorders.
To read the paper, please visit: https://journals.sagepub.com/doi/10.1177/22143602251399244
Joost Kools, Lawrence Korngut, Janet Petrillo Ballantyne, Irene Roozen, Ria de Haas, Amanda Hill, Teresinha Evangelista, Valeria A Sansone, Richard Roxburgh, Hanns Lochmuller, Jeff Statland, Nicholas E Johnson, and Nicol Voermans Journal of Neuromuscular DiseasesOnlineFirst © The Author(s) 2025