P139 Correlation and validation of the North Star Ambulatory Assessment, timed test and motor function measure centiles for boys with Duchenne muscular dystrophy

There is significant heterogeneity in the North Star Ambulatory Assessment (NSAA) trajectories of boys with DMD, which may present a challenge for clinical care and development of clinical trials. Centiles quantify the cross-sectional variability in cohorts, are easily interpretable for patients and parents, and have been described for the Motor Function Measure (MFM) in boys both on and off corticosteroids (CS). We used the NorthStar Database to develop NSAA, Rise from Floor (RFF) and 10m Walk (10MWR) centiles-for-age for boys with DMD on CS.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: E. Milev, G. Stimpson, M. van der Holst, A. Wolfe, E. O'Reilly, A. Manzur, E. Niks, S. Houwen-Opstal, G. Baranello, F. Muntoni Source Type: research