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Clinical variability in 48 steroid-naïve Duchenne muscular dystrophy boys, age 4 to < 7 years. Shown is Time to Stand velocity (1/event) in DMD boys enrolled into vamorolone clinical trials. Extensive variability in disease severity is seen, with a 6-fold difference in velocity from the most severe to the mildest patient (from Dang et al., 2020 and Smith et al., 2020, mod.) 38,39.
