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Strategies to prevent transthyretin (TTR) amyloidosis. Liver transplant is the standard practice since TTR (an amyloidogenic variant in familial amyloid polyneuropathy (FAP) patients) is mainly produced in the liver. Tegsedi inhibits hepatic production of TTR (both wild-type and amyloidogenic variants). TTR kinetic stabilizers are compounds that selectively bind to TTR, preventing its dissociation (Tafamidis was approved in E.U.).
