Drug to treat all genetic variants of DMD


United States

A drug for the treatment of patients with Duchenne muscular dystrophy (DMA) was approved by the US Food and Drug Administration on 22 March. Duvyzat (givinostat) is the first nonsteroidal drug for patients with all genetic variants of the disease. DMD is a rare neurological disorder which causes progressive muscle weakness due to a lack of dystrophin, a muscle protein. Over time, individuals with the disease experience muscle weakness that can lead to problems with walking, breathing and an early death.