Fordadistrogene movaparvovec for Duchenne muscular dystrophy

Fordadistrogene movaparvovec is a medicinal product in clinical development for the treatment of ambulatory boys with a confirmed genetic diagnosis of Duchenne muscular dystrophy (DMD). DMD is a severe and rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused by an absence of functional dystrophin, a protein that helps keep muscle cells intact. It affects mainly boys and symptoms often start before the age of five. It causes progressive muscle weakness and often leads to loss of walking ability by the age of twelve, as well as problems with the heart and lungs. The main symptom is muscular weakness, with other common symptoms including enlargement of the calves, a waddling gait, lumbar lordosis, running, jumping, and walking difficulties. There is no cure for DMD, and treatment is mainly supportive, with mortality primarily due to cardiac and/or respiratory complications in their 20s or 30s.