Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
Hereditary transthyretin (ATTR variant) amyloidosis, also called hATTR amyloidosis is a rare, rapidly progressive, debilitating, and fatal disease caused by transthyretin (TTR) gene variants. Misfolded TTR accumulates as amyloid