Duchenne muscular dystrophy (DMD) is a genetic disease in which patients lack the protein dystrophin, which is critical for stabilizing muscle cell membranes. Without a functional dystrophin protein, muscle cells are susceptible to damage and progressively die. Patients experience progressive muscle weakness starting at an early age, loss of ambulation typically in the first decade of life, eventual respiratory and cardiac failure, and an abbreviated lifespan averaging less than three decades.
Approximately 15,000 to 20,000 boys and young men are living with the disease in the United States1. The annual cost of care for patients with DMD is very high and increases with disease progression. We, therefore, believe that DMD represents a significant market opportunity for our lead product candidate. CAP-1002 has been granted orphan drug designation by the FDA for the treatment of DMD.
Click here to view a webcast on cardiosphere-derived cells for heart failure in Duchenne muscular dystrophy by Eduardo Marbán, M.D,Ph.D. – Director, Cedars-Sinai Heart Institute
1 Duchenne Muscular Dystrophy United States Prevalence Estimate