Transthyretin amyloid cardiomyopathy (ATTR-CM) was previously considered an inescapable progressive disease leading to devastating outcomes. Clinicians often disregarded ATTR-CM, as the therapeutical arsenal available was very limited. Historically, treatments have offered mostly symptomatic relief without altering disease progression. Fortunately, the last decade brought us disease-modifying treatments with the potential to transform the course of ATTR-CM. At a molecular level, ATTR-CM develops when transthyretin (TTR), usually a stable transport protein for thyroxine and vitamin A, dissociates from its tetrameric configuration into […]

Patisiran is an RNA interference therapeutic agent used in the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis), a condition characterized by the accumulation of misfolded transthyretin (TTR) protein amyloid fibrils in tissues.– As shown in , the mechanism of action differs from previous drugs such as tafamidis, which promotes the stabilization of the TTR protein, rather than the synthesis inhibition., Patisiran uses a mechanism involving small interfering RNA to specifically target the messenger RNA (mRNA) responsible for producing the TTR protein […]