Work

Patisiran in the Treatment of Cardiac Amyloidosis: Good for the World and Good for Brazil

Year: 2025

Type: article

Abstract: 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 [...]

Source: Arquivos Brasileiros de Cardiologia

DOI: 10.36660/abc.20250335i

Author: Humberto Villacorta

Institution: Universidade Federal Fluminense

Language: English

Topic: Amyloidosis: Diagnosis, Treatment, Outcomes

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: diamond

OpenAlex ID: W4412722081