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Duchenne muscular dystrophy (DMD) is a sex-linked genetic disorder that invariably causes progressive weakness and death. Any treatment that improves the prognosis should be welcome, but the first report of the beneficial effects of steroids in DMD (Lancet 1974; 304:1409) was met with skepticism (Lancet 1975; 305:276), requiring decades before being accepted as the standard of care (Neuromuscular Disorders 1991; 1:161; NEJM JW Neurol Feb 2018 and Lancet 2018; 391:451). Corticosteroids delay wheelchair requirement by up to 4 years, improve quality of life, and extend survival. However, potential adverse effects of corticosteroids — weight gain, osteopenia, growth impairment, and behavioral disturbances — have raised concerns.
In this manufact…