Coelho et al., NEJM. 2013 Sept13 (Epub doi: 10.1056/NEJMoa1208760 )
Abstract
Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin