Duchenne muscular dystrophy
Introduction
Duchenne Muscular Dystrophy (DMD) is one of the most common and severe forms of muscular dystrophy affecting children. DMD is an X-linked condition, meaning that it usually only affects boys. The cause of DMD is a mutation in the gene that produces a protein called dystrophin. Dystrophin acts as a shock absorber, protecting the muscles from injury. Without dystrophin, muscle cells are not able to repair themselves and they start to degenerate. This leads to loss of muscle and muscle strength.
Summary
The Share4Rare team has worked with clinical experts, patients and caregivers, to produce information for families affected by DMD. You will learn more about the causes of DMD, signs and symptoms, diagnosis, treatment and therapies, clinical trials and disease management. Our goal is to produce accessible information to help families become experts in their condition.
