Source
National Library of Medicine
Hereditary transthyretin‐mediated amyloidosis (hATTR) is a disorder that affects several body systems and can result in life‐threatening conditions like cardiomyopathy and polyneuropathy. For treatment of hATTR in the new medical realm, there is a breakthrough discovery of a medicine called Eplontersen, which targets the underlying genetic mutation causing the condition. This review aims to summarize Eplontersen's mechanism of action, efficacy, safety profile, and ongoing clinical trials.
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