These are peer-reviewed supplementary data for the article 'Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA' published in the Journal of Com
Objective: We carried out first-in-patient studies of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. Methods: An open-label, multiple-asce
