Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy
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BackgroundDuchenne muscular dystrophy (DMD) leads to dilated cardiomyopathy and heart failure during teenage years or young adulthood. Eteplirsen promotes dystrophin production through skipping of exon 51 of the DMD gene.ObjectiveThis analysis compared LVEF de... ...