Duchenne Muscular Dystrophy

April 25th 2024

The study provides evidence of survival benefits among patients with Duchenne muscular dystrophy (DMD) receiving eteplirsen compared with the natural history of the condition.

April 19th 2024

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.

April 12th 2024

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).

March 21st 2024

The approval follows fast track, orphan drug, and rare pediatric designations and an original Prescription Drug User Fee Act date of December 21, which the FDA pushed back to March 21 to have more time to review Italfarmaco’s application.