Duchenne Muscular Dystrophy-associated Cardiomyopathy

Duchenne Muscular Dystrophy (DMD) is a genetic disease in which patients lack a functional dystrophin protein, a protein critical for stabilizing muscle cell membranes. Patients experience progressive muscle weakness starting at an early age, loss of ambulation in the first decade of life, eventual respiratory and cardiac failure, and an abbreviated lifespan averaging less than three decades. Without a functional dystrophin protein, heart muscle cells are susceptible to damage and progressively die. This amplifying process triggers progressive scar tissue deposition and leads eventually to heart failure. Improvements in the treatment of respiratory muscle disease have elevated the almost inevitable cardiomyopathy to the leading cause of death in DMD patients. Nearly 20,000 boys are living with the disease in the United States alone and approximately 275,000 worldwide are affected. No specific therapies currently exist to treat DMD cardiomyopathy. Capricor has announced plans to pursue a clinical program for the treatment of DMD-associated cardiomyopathy using its lead product candidate, CAP-1002.