Loading...
Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disease caused by mutations in the dystrophin gene, which lead to progressive muscle degeneration and weakness. The mainstay of treatment has been corticosteroids and, more recently, gene therapy targeting certain DMD genetic variants.
Givinostat is a pan-histone deacetylase inhibitor that counteracts downstream effects of dystrophin deficiency, reducing inflammation and fibrosis. Based on the results of this industry-funded, multicenter, randomized, double-blind trial, on March 21, 2024, the FDA approved givinostat (Duvyzat) for patients 6 years of age and older with DMD. Ambulatory male children 6 years of age or older (mean age, 9.8 years) with genetically confirmed DMD on a s…