Duchenne muscular dystrophy (DMD) is a genetic disease that weakens muscles and often affects the heart. Heart disease is a leading cause of serious illness in people with DMD.
A study published in Molecular Therapy tested the experimental drug Setanaxib in two preclinical models of DMD. The treatment preserved cardiac function, limited heart enlargement and reduced tissue scarring. The findings also point to the enzyme NOX4 as a possible target for future treatments of cardiomyopathy, a serious heart condition linked to DMD.
Setanaxib works to limit oxidative stress and lowered inflammation and fibrosis in the models. The researchers hope the drug will be tested soon in clinical trials for people with DMD.
Difficult words
- genetic — Relating to genes and inherited from parents
- enzyme — A protein that helps chemical reactions in body
- cardiomyopathy — A disease of the heart muscle causing weakness
- inflammation — Body reaction that causes redness and swelling
- fibrosis — Thick or scar tissue that forms in organs
- preclinical — Before tests in people; in lab or animals
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Discussion questions
- How could a drug that protects the heart help people with DMD?
- What would you want to know before trying a new drug in a clinical trial?
- Why is it important to test a drug first in models before people?
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