Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (also referred to as Duchenne) is a rare, life-shortening genetic disorder that affects boys and causes their muscles to break down and lose strength over time. Duchenne is caused by specific genetic mutation (error) in the gene that codes for dystrophin. Dystrophin is a protein that plays a key role in the function of muscle cells and protects them from damage as muscles contract and relax. These mutations in the dystrophin gene lead to a lack of dystrophin protein in muscles. Without enough dystrophin, muscles gradually grow weaker until they can’t move at all, and eventually breathing and heart function are lost.