Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls

Francesco Muntoni, James Signorovitch, Gautam Sajeev, Nathalie Goemans, Brenda Wong, Cuixia Tian, Eugenio Mercuri, Nicolae Done, Hallee E. Wong, Jackson Moss, Zhiwen Yao, Susan J. Ward, Adnan Manzur, Laurent Servais, E. Niks, Volker Straub, Imelda JM de Groot, Craig M. McDonald