Patisiran in hereditary transthyretin-mediated amyloidosis

摘要

The clinical course of hereditary transthyretin-mediated amyloidosis has substantially changed in the past 10 years.1 Several clinical studies have widened the therapeutic landscape for the condition, including transthyretin (TTR) stabilisers and TTR gene silencers. The use of liver transplantation, which, until a few years ago, represented the only therapeutic choice, is now declining.1 Furthermore, new monoclonal antibodies targeting TTR deposits could be tested in future clinical trials.1