DMD Preclinical Program

About DMD

Duchenne muscular dystrophy (DMD) is a degenerative neuromuscular disease that leads to progressive weakness in skeletal muscles and heart. DMD is caused by certain loss-of-function mutations in the dystrophin gene (DMD), and it follows an X-linked, recessive pattern of inheritance. In the absence of functional dystrophin protein, muscles sustain ongoing damage and deterioration marked by limited muscle growth, strength, and flexibility, and there are additional adverse impacts on respiratory and cardiac function. DMD mostly affects males, and the global incidence of DMD is approximately 1:5,000 live male births.

DMD is the most common type of muscular dystrophy worldwide. It is typically diagnosed between the ages of 2 and 5, and it often presents with developmental delays in sitting, walking, and speech though progression of symptoms varies for each patient. Patients experience progressive loss of ambulation at ~12 years of age, and median survival is 25-30 years.