Duchenne muscular dystrophy (DMD) is a devastating genetic disorder, typically affecting males, and is characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD lack a muscle protein called dystrophin, and they typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30.
Roughly 200,000 boys and young men around the world are affected by DMD. Although there is no cure for this childhood form of muscular dystrophy, there are several approved drugs to treat this condition. The approved drugs include Sarepta Therapeutics' Exondys 51, Vyondys 53, Amondys 45, and Elevidys; PTC Therapeutics' Emflaza; NS Pharma's Viltepso; and Italfarmaco Group's Duvyzat.
For comments and feedback: editorial@rttnews.com