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