TFS's Strategy For Addressing Site Selection Challenges In DMD Gene Therapy Studies

This case study examines the challenges associated with site selection in clinical trials for Duchenne muscular dystrophy (DMD) gene therapy and highlights a strategic approach to addressing these obstacles. DMD, a rare and progressive neuromuscular disorder, presents unique complexities in trial design due to its patient demographics and disease heterogeneity. Effective site selection is critical for the success of trials but often hindered by limited site experience, patient access constraints, and regulatory nuances. The study details innovative methodologies employed to streamline site identification, leveraging advanced analytics, centralized data platforms, and cross-disciplinary collaboration. These strategies enhance site readiness, ensure regulatory compliance, and optimize patient recruitment pathways. The findings underscore the importance of a tailored approach to trial execution in rare diseases and demonstrate how addressing logistical and operational barriers can accelerate therapeutic development in the field of gene therapy for DMD.