Consistency of changes in %-predicted forced vital capacity between real-world data and trial placebo arms in ambulatory Duchenne muscular dystrophy
Consistency of changes in %-predicted forced vital capacity between real-world data and trial placebo arms in ambulatory Duchenne muscular dystrophy
To assess risk of bias in the use of real-world and natural history data (RWD/NHD) as external controls, this study compared change in pulmonary function, as measured by percent-predicted forced vital capacity (FVC%p) between RWD/NHD sources and trial placebo arms in ambulatory boys with DMD.
This poster was presented at WMS 2022 and MDA 2023.
Authors: N. Goemans, C. McDonald, F. Muntoni, J. Signorovitch, G. Sajeev, N. Done, A. Manzur, B. Wong, C. Tian, E. Mercuri, C. He, D. Peterson, H. Akbarnejad, S. Ward, PRO-DMD-01 study investigators, and cTAP.