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Cardiac TTR Amyloidosis Therapies
Gene silencing therapy, using a silencing RNA packaged in lipid nanoparticle and delivered to the liver, has been shown to be effective at treating the polyneuropathy associated with transthyretin (TTR) amyloidosis. Stabilization of the TTR tetramer with tafamidis is effective in treating both polyneuropathy and cardiomyopathy. Monoclonal antibodies to extract TTR amyloid deposits from tissues are in clinical trials. Figure created with biorender.com.
