Researchers report a possible way to protect the heart in people with Duchenne muscular dystrophy (DMD), a progressive genetic disease that weakens muscles and often affects the heart. The study appears in the journal Molecular Therapy and used two preclinical DMD models to test an experimental drug called Setanaxib.
The treatment preserved cardiac function, limited enlargement of the heart and reduced tissue scarring. Setanaxib targets enzymes called NOX1 and NOX4 to limit oxidative stress. In the study it reduced inflammation and fibrosis and lowered activity of genes linked to cardiomyopathy while helping the heart keep its pumping ability.
The research was led by Da-Zhi Wang and included John Mably, Gabriela Diniz and outside collaborators. The team hopes the Nox4 inhibitor will be tested in clinical trials to slow the progression of heart disease in DMD patients. The work builds on more than 15 years of research in Wang’s laboratory.
Difficult words
- progressive — becoming worse or more serious over time
- preclinical — done before tests in people
- preserve — keep something in the same conditionpreserved
- oxidative stress — damage caused by reactive oxygen molecules
- inflammation — body response that causes redness and swelling
- fibrosis — formation of excess scar tissue in organs
- cardiomyopathy — disease that weakens the heart muscle
- inhibitor — substance that slows or stops a process
Tip: hover, focus or tap highlighted words in the article to see quick definitions while you read or listen.
Discussion questions
- Why is protecting the heart important for people with Duchenne muscular dystrophy?
- What should researchers do next before doctors can use Setanaxib for patients?
- How could lowering inflammation and fibrosis help a person with heart disease in daily life?
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