What question did this study set out to answer?

The study aims to evaluate the effectiveness of eplontersen in improving outcomes for patients with hereditary transthyretin amyloidosis with neuropathy, irrespective of sex.

April 8, 2026Open Access

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients

Key Points

The study aims to evaluate the effectiveness of eplontersen in improving outcomes for patients with hereditary transthyretin amyloidosis with neuropathy, irrespective of sex.
Exploratory analysis of treatment effects of eplontersen versus placebo
Assessment of neuropathy impairment and quality of life measures
Included both male and female patients, considering those with cardiomyopathy
Eplontersen halted neuropathy impairment in patients
Improvement in quality of life was observed in both sexes
Effectiveness was similar between male and female patients

Abstract

Eplontersen halted neuropathy impairment and improved quality of life versus placebo to a similar degree in both male and female patients with ATTRv-PN, including those with cardiomyopathy. These findings support the use of eplontersen as an effective treatment for ATTRv-PN regardless of sex.

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients

Key Points

Abstract

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