P131 MRI fat fraction distribution in Duchenne muscular dystrophy (DMD): effect size comparison to identify optimal biomarker for early efficacy assessment

Changes in mean MRI proton density fat fraction (MRI-PDFF) in DMD and other neuromuscular trials are assessed as a potential early biomarker of efficacy. However, mean MRI-PDFF within the muscle region of interest (ROI) may not reflect pathologically relevant changes in the distribution of fatty infiltration with disease modifying treatments. We hypothesized that other biomarkers indicative of changes in the distribution may have a larger effect size (Cohen's d) thereby increasing statistical power to detect efficacy if utilized in clinical trials.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: M. Hammond, J. Harris, B. Luna, F. Roche, F. Vincent, M. Berger, S. Zabbatino, R. Scheyer, S. Holland Source Type: research