Duchenne muscular dystrophy (DMD) is a genetic disorder in which the body lacks dystrophin, a protein that helps keep muscle cells intact. It causes muscles to rapidly degenerate and weaken. DMD predominantly affects males. A majority of patients develop heart or respiratory failure, surviving into their 20s or early 30s.
Subha Raman, a cardiologist and professor at the Ohio State University's Wexner Medical Center, led a team in a clinical trial that tested the combination of eplerenone and either an ACE inhibitor or an angiotensin receptor blocker (ARB) to decrease the progression of heart muscle disease - a leading cause of death in boys and young men with DMD.
"We believe this research offers evidence that supports the early use of these readily available medications," said Raman. Raman and her team based this trial on their earlier lab findings that showed this combination of medicines reduced muscle damage and preserved function in an animal model of DMD.
In this trial, researchers enrolled 42 boys with DMD who also showed evidence of early heart muscle damage by cardiac magnetic resonance imaging.In the double-blind study, the boys were randomized to receive one pill of either 25 mg of eplerenone or placebo daily for one year.The study was published in the journal The Lancet Neurology.