DMD is characterized by progressive muscle degeneration and weakness. caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The disease primarily affects boys, but in rare cases, it can affect girls.
Symptoms begin to manifest themselves usually between ages 3 and 5, initially with muscle weakness of the hips, pelvic area, thighs and shoulders, and progress to the skeletal (voluntary) muscles in the arms, legs, and trunk. By the early teens, the heart and respiratory muscles also are affected.
At Evox we work with a UK-based charity, Duchenne UK, with the aim to develop a novel therapeutic strategies for DMD based on replacing dystrophin.