The U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
DMD is the most common childhood form of muscular dystrophy and typically affects males. It is a rare neurological disorder which causes progressive muscle weakness due to a lack of muscle protein called dystrophin. Over time, the muscles deteriorate causing problems with walking and muscle strength and ultimately problems with breathing leading to early death. Life expectancy for those with DMD has increased over the years, with some patients surviving beyond 30 years.
