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Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy
Date:6/26/2012

20s.

Still, there remains no consensus on how to best manage DMD-associated heart fibrosis and disease, Hor said. Often, between the ages of 7 and 10, echocardiogram results

show the heart is functioning normally by conventional measures delaying needed heart treatment in an intensely time-sensitive disease.

Evidence-driven approach

Hor and his colleagues theorize earlier diagnosis and treatment with anti-fibrotic medication could enhance quality of life and survival for people with DMD. They base this on a broad range of studies at Cincinnati Children's and other institutions, including reviews of clinical data.

One stark conclusion from those reviews is that, by the time current diagnostic methods catch DMD-associated heart disease after the age of 10, damage is often irreversible. By then, standard treatment with beta-blockers and angiotensin converting enzyme inhibitors provide little if any long-term benefit.

One study co-authored by Hor reviewed the cases of 247 children with DMD. Thirteen percent of patients under the age of10 years already had significant evidence of cardiac scarring even though conventional tests indicated normal heart function.

Standard echocardiograms now used in DMD look for declines in left ventricle ejection fraction (LVEF) a measure of heart muscle pumping ability. Although reliable for many heart conditions, functional assessments by echocardiograms are not sensitive enough to detect early DMD-associated heart disease, such as heart muscle fibrosis, scarring and important functional decline that precedes measurable LVEF reductions.

To overcome this limitation, the clinical trial is using advanced cardiac magnetic resonance imaging (CMR) techniques. They include imaging with a contrast agent called gadolinium, which allows physicians to detect the early formation of heart muscle fibrosis while patients are still young and ejection fraction is n
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Contact: Nick Miller
nicholas.miller@cchmc.org
513-803-6035
Cincinnati Children's Hospital Medical Center
Source:Eurekalert

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