Breakthrough in Rare Diseases: FDA Approves Givinostat for Duchenne Muscular Dystrophy

A major development in the treatment of a rare genetic disease, duchenne muscular dystrophy, has recently been approved by the FDA. Givinostat, a histone-acetylase inhibitor, signifies a new ray of hope for patients with DMD, a progressive and enervating disease. This treatment, given as an oral suspension, was approved for use in patients 6 years and older after promising results were obtained from the phase 3 EPIDYS trial. Givinostat can also cause adverse effects such as thrombocytopenia and hypertriglyceridemia, and should be administered with continuous monitoring.