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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
- All authors
- Muntoni, F.; Signorovitch, J.; Sajeev, G.; Goemans, N.; Wong, B.; Tian, C.X.; Mercuri, E.; Done, N.; Wong, H.; Moss, J.; Yao, Z.W.; Ward, S.J.; Manzur, A.; Servais, L.; Niks, E.H.; Straub, V.; Groot, I.J. de; McDonald, C.; North Star Clinical Network PRO-DM; Assoc Francaise Myopathies AFM; DMD Italian Grp; Collaborative Trajectory Anal Proj
- Date
- 2022-04-01
- Journal
- Neuromuscular Disorders
- Volume
- 32
- Issue
- 4
- Pages
- 271 - 283