givinostat

Givinostat is a small molecule histone deacetylase (HDAC) inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. It is the first non-steroidal drug approved to treat all genetic variants of DMD. Givinostat works by inhibiting HDAC enzymes, which are overactive in DMD and contribute to inflammation and impaired muscle repair. By blocking these enzymes, givinostat may reduce inflammation, slow muscle breakdown, and support muscle regeneration. The precise mechanism by which it benefits patients with DMD remains unknown but is believed to involve upregulation of follistatin and stimulation of myogenesis[1][2][5][7][9]. Givinostat has also been investigated for other conditions such as polycythemia vera, Crohn's disease, juvenile idiopathic arthritis, leukemia, lymphoma, and other muscular dystrophies[5][6].

Brand names

Duvyzat

Other names

givinostat hydrochloride monohydrategivinostat hydrochloride