Abstract
Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, debilitating disease. Orthotopic liver transplant (OLT) eliminates variant transthyretin (TTR) to slow progression in early-stage hATTR amyloidosis. Patisiran reduces production of variant and wild-type TTR, as shown in patients who had not undergone OLT in the APOLLO study.
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