Precision medicine with pioneering RNAi therapeutics in ATTR amyloidosis: from bench to bedside.
Transthyretin (ATTR) amyloidosis is a multisystem disease caused by misfolding and aggregation of the plasma protein transthyretin (TTR) into insoluble amyloid fibrils in various organs. In clinical practice, ATTRwt amyloidosis manifests as a predominant cardiomyopathy (ATTR-CM)…