The study provides evidence of survival benefits among patients with Duchenne muscular dystrophy (DMD) receiving eteplirsen compared with the natural history of the condition.
A recent study suggests the differences between ambulation definitions for patients with Duchenne muscular dystrophy (DMD) can impact the identification of ambulant vs nonambulant individuals, and standard criteria across settings are needed.
A recent study suggests treatment with glucocorticoids after loss of ambulation can preserve late-stage functional abilities, respiratory function, and cardiac function in patients with Duchenne muscular dystrophy (DMD).