1 2 aim

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

Goemans N, Signorovitch J, Sajeev G, Yao Z, Gordish-Dressman H, McDonald CM, et al. Suitability of external controls for drug evaluation in Duchenne muscular dystrophy. Neurology. 2020;95(10):e1381-e91   Abstract OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory Duchenne muscular […]