P138 Development and evaluation of a composite time-to-progression endpoint that spans ambulatory and non-ambulatory stages of Duchenne muscular dystrophy (DMD)

This study developed and characterized a composite endpoint for clinically meaningful progression in DMD across three domains: ambulatory motor, upper-limb motor, and pulmonary function. Progression criteria were evaluated in each domain: A) loss of ≥ 3 NSAA total score points; B) loss of ≥ 3 PUL1.2 total score points; C) decline of FVC %-predicted [FVC%p] ≥ 10% points.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: C. McDonald, F. Muntoni, J. Marden, N. Goemans, A. Gomez-Lievano, A. Zhang, Z. Chen, S. Ward, J. Signorovitch Source Type: research