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Inhibiting NF-κB improves heart function in Duchenne muscular dystrophy

August 31, 2018 / Drug Target Review
SHARESHARESHARE

Duchenne muscular dystrophy (DMD) is a devastating genetic disease that impairs cardiac and skeletal muscle development. People with DMD gradually lose ambulation in childhood, acquire respiratory and heart failure in young adulthood and succumb to the disease by their mid-thirties. Until recently, there has been no effective treatment for the characteristic muscle-wasting progression of this disease. Provisional FDA approval of the first DMD therapy (eteplirsen) and improved disease management strategies have extended the lifespan of DMD patients and expanded the field of DMD research into later-stage outcomes such as cardiomyopathy (heart failure).