Givinostat hydrochloride is primarily used in the treatment of Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene leading to deficient or dysfunctional dystrophin protein. It works as a histone deacetylase (HDAC) inhibitor, which helps regulate gene expression and promotes muscle regeneration. This compound has shown potential in reducing inflammation and fibrosis in muscle tissues, improving muscle function and strength in patients. It has been approved for use in DMD in certain regions. Additionally, it is being investigated for its therapeutic effects in other conditions such as polycythemia vera, a type of blood cancer, where it helps control excessive red blood cell production. Its ability to modulate gene expression makes it a promising candidate for various other genetic and inflammatory diseases.