DMD associated cardiomyopathy is a type of heart disease that is caused by mutations in the DMD gene and consequent lack of dystrophin. The heart becomes scarred and weakened and does not pump blood around the body efficiently. Around 90% of people with Duchenne develop cardiomyopathy.

Management of cardiomyopathy involves diagnosis and treatment as early as possible. Several drugs are currently available and include angiotensin converting enzyme (ACE) inhibitors such as enalapril or lisinopril, and angiotensin receptor blockers (ARBs), such as losartan. These drugs relax the arterioles around the heart so lessening the work load on the heart.

Beta-blockers, such as bisoprolol or carvedilol, may be added, as they slow down the heart rate allowing the heart to work more efficiently.

Diuretics, such as spironolactone, help the body to remove water so the volume of blood to be pumped is less, they may be added when there is evidence of heart failure.

Anti-arrhythmic therapy is also sometimes needed. Left ventricular assist devices (LVADs) may increasingly be offered to boys with Duchenne that are in heart failure.

There is also recent evidence suggesting that prolonged steroid therapy may be helpful in delaying the onset of cardiomyopathy.

Capricor Therapeutics is developing a therapy, CAP-1002, which is currently in phase I/II trials. The investigational therapy is an allogeneic, off-the-shelf “ready to use” cardiac cell therapy which comes from donor heart tissue and is infused directly into a patient’s coronary artery. CAP-1002 is being tested to determine if it is safe and effective in impacting heart function and structure.