Abstract
Hereditary transthyretin-mediated (hATTR) amyloidosis, also known as ATTRv amyloidosis, is a progressive disease caused by misfolded transthyretin (TTR) that accumulates as amyloid fibrils in multiple tissues and organs. Vutrisiran, an investigational RNAi therapeutic for the treatment of ATTR amyloidosis, targets variant and wild-type TTR.
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