Randomized Trialhigh evidence

Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial.

Nature medicine·August 30, 2026·PMID 42742182

Why it matters

The choice of background therapy may determine whether adding a TTR gene silencer provides incremental benefit in ATTR-CM. This analysis suggests that patients not already on a stabilizer are most likely to benefit from eplontersen, whereas those on stabilizers may not gain additional protection.

Infographic summary of Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial.
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