Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy.

J Neuromuscul Dis · 2026 · PMC13141851 · PMID 40831143

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highly significantno p-value reported

33 This is particularly true when the clinical effect is considered highly significant, as was the case in this study, with the observed differences in LVEF decline, especially below the 50% threshold.

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