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Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Hoffman EP, Schwartz BD, Mengle-Gaw LJ, et al. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 2019;93:e1312-e1323   Abstract OBJECTIVE: To study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne muscular dystrophy (DMD). METHODS: An open-label, multiple-ascending-dose study of vamorolone was conducted in 48 boys with DMD (age 4-<7 years, […]