Duchenne Muscular Dystrophy (DMD) Treatment: An Informational Overview
Introduction
Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder that primarily affects boys and causes progressive muscle weakness. It results from mutations in the dystrophin gene, which leads to a lack of dystrophin protein—a critical component needed for maintaining the structural stability of muscle cells. Over time, muscle degeneration leads to loss of mobility and can affect respiratory and cardiac muscles.
What Duchenne Muscular Dystrophy Is
DMD is an inherited condition that typically becomes noticeable in early childhood, often between ages 2 and 5. Children with DMD may show delayed motor development, frequent falls, difficulty running or climbing stairs, and enlarged calf muscles due to muscle tissue being replaced by fat and connective tissue. Because the condition is progressive, symptoms worsen over time.