Validation of a composite prognostic score for time to loss of ambulation in Duchenne Muscular Dystrophy
Validation of a composite prognostic score for time to loss of ambulation in Duchenne Muscular Dystrophy
An independent validation of a previously developed composite prognostic score for LoA using data the CINRG Duchenne natural history study, which was not used for score development.
This poster was presented at WMS 2022 and MDA 2023.
Authors: C. McDonald, H. Gordish-Dressman, J. Signorovitch, G. Sajeev, M. Fillbrunn, M. Frean, S. Ward, N. Goemans, K. Vandenborne, E. Mercuri, F. Muntoni, Investigators for CINRG-DNHS, The PRO-DMD-01 Study, The Imaging DMD Study, The iMDEX Study, and cTAP.