Study offers hope for Duchenne muscular dystrophy patients

Dont underestimate a fathers determination.

Ty Ballou of Ben Avon was eager for a new treatment for his son Ryan, now 27, who has Duchenne muscular dystrophy, uses a wheelchair and already has outlived what his life expectancy would have been.

After helping to inspire a researcher to test a new drug regimen, he raised nearly $250,000 over six years to fund research at the Ohio State University Wexner Medical Center. He collected contributions to complete marathons and other distance races, raised money from the sale of Ballou Skies peanut butter for two years at Giant Eagle and continues operating the web site http://www.ballouskies.com to seek donations and provide information about the disease that predominantly affects males.

The results are in.

A study published today in The Lancet Neurology and led by Subha Raman, a cardiologist and professor at the medical center, shows that a combination of prescription drugs was used successfully to reduce the progression of heart failure in those with Duchenne.

Those results follow Mr. Ballous sons success with a similar regimen of drugs hes been taking for seven years. Those include the ACE inhibitor lisinopril and the diuretic spironolactone, which typically are used to treat advanced heart failure. Ryan has experienced no heart deterioration since he began taking the drugs.

Its amazing, Ryan said of the study he helped to inspire. This might be something that can make a significant change in care.

The study used a similar combination of drugs including the diuretic eplerenone with an ACE inhibitor or an ARB (angiotensin receptor blocker) to decrease the progression of heart-muscle disease, which is a leading cause of death in boys and young men with Duchenne, usually by the time they reach their 20s or early 30s.

The double-blind study involved 42 patients with the condition. Ryan did not participate but undergoes an annual MRI each December.

After 12 months of treatment, the team reported that patients taking the drugs showed significantly less decline in left ventricular function than those patients on placebo, an Ohio State release states. Six months to a year of treatment is necessary before patients experience benefits.

Read the rest here:
Study offers hope for Duchenne muscular dystrophy patients