Research links Duchenne muscular dystrophy with heart failure

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Imagine being told you won’t survive past your mid-20s. This is a reality for many young men with Duchenne muscular dystrophy (DMD) – a devastating disease that causes muscle breakdown and eventually heart failure in boys and young men.

DMD is an inherited genetic disorder that causes muscles to rapidly degenerate and weaken. The majority of DMD patients develop heart or respiratory failure, and don’t survive past their 20s or early 30s.

The leading cause of death in patients with Duchenne muscular dystrophy is heart muscle disease. A new study led by Subha Raman, MD, a cardiologist at The Ohio State University Wexner Medical Center, shows that early use of heart failure drugs slows the decline in heart function in DMD patients.

The inspiration behind the research, 27-year-old Ryan Ballou, is a DMD patient who was diagnosed more than 23 years ago. Ryan’s been coming to Ohio State's Wexner Medical Center for treatment for more than 23 years. Five years ago, Ryan’s neurologist referred him to Ohio State’s Heart and Vascular Center, where a team is collaborating on a revolutionary drug treatment that may help delay heart failure in DMD patients.

Ryan, along with his father, started an organization called BallouSkies to raise money for Raman’s research and was the primary financial supporter of this clinical study.

“Research progressed much faster thanks to their support,” says Raman. “The work we have accomplished in just a few years would have taken a decade or more if we had to seek traditional funding.”

Raman’s research shows that when certain heart failure drugs are given to patients with DMD early on, the progressive decline in heart function slows before symptoms present. Early treatment with lisinopril and spironolactone significantly preserves heart and skeletal muscle tissue.

Since Ryan started taking the medication over eight years ago, there hasn’t been any more deterioration of his heart muscle. “I’ve been ahead of the curve,” Ryan says. “I walk longer than most people with Duchenne’s and I still have a lot of strength in my arms.” Ryan's success holds promise for thousands of men affected by DMD.

At this time, treatment begins after heart damage is found in DMD patients, but the team suggests that using the drugs sooner could prevent damage before it starts. “Despite the inevitable muscle deterioration seen in Duchenne patients, this drug combination provides nearly normal muscle function and considerable preservation of tissue,” says Raman. “We believe this research offers evidence that supports the early use of these medications,” says Raman. This could quickly become the standard of care for DMD patients. If these results translate into future clinical trials, it could mean that DMD patients may never have to use wheelchairs and could live long and happy lives.

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