CPN | Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Taken from Duchenne.Com

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is one of four conditions known as dystrophinopathies. 

DMD primarily affects boys, but in rare cases, it can affect girls.  Symptom onset is in early childhood, usually between ages 2 and 3..and the affected child might have difficulty jumping, running, and walking. Later on, the heart and respiratory muscles are affected as well.

Until relatively recently, children with DMD usually did not survive much beyond their teen years. Advances in cardiac and respiratory care have increased the projected life expectancy and many affected individuals can live into their early 30’s. There is currently no cure for DMD. However, two mutation-specific treatments have been approved by the FDA. 

For more information, visit the Muscular Dystrophy Association

Note: As science advances, we continue to experience a fast-evolving landscape of potential new therapies. The families portrayed here offer a perspective on coping with rare and/or life-limiting conditions. They also each represent a particular moment in time.

If you are considering a clinical trial for your child, please also see the Courageous Parents Network Guided Pathway on Clinical Trials.