Transstyretin amyloidosis (ATTR) is a minor, progressive, and deadly disease caused by extracellular deposits of amyloid fibers composed of transthyretin (TTR) in various organs. Under normal conditions, TTR is a protein that acts as a transporter of thyroxine and vitamin A and is mostly synthesized in the liver (95%). TTR circulates in the form of a tetramer, but certain mutations cause its destabilization and the formation of monomers that fold abnormally and accumulate in different organs and tissues (mainly in the peripheral nervous system and heart) where it causes a progressive dysfunction.
Tafamidís; Inotersen; Patisiran, Hereditary transthyretin amyloidosis; Polyneuropathy
Programa d'Harmonització Farmacoterapèutica. Tafamidís, inotersèn i patisiran per al tractament de l'amiloïdosi transtiretina hereditària. Barcelona: Servei Català de la Salut; 2020.
Use this identifier for quote and/or link this documenthttps://hdl.handle.net/11351/5465
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