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Patisiran for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis
source: Expert Review of Clinical Pharmacology
year: 2019
authors: Yang J
summary/abstract:Hereditary transthyretin-mediated amyloidosis is caused by a mutation in transthyretin (TTR) gene resulting in misfolded TTR protein accumulating as amyloid fibrils. Patisiran is a lipid nanoparticle formulation of ribonucleic acid interference (RNAi), which can reduce the production of TTR. Areas covered: In this review, the chemical property, mechanism of action, pharmacokinetics, clinical efficacy, and safety of patisiran were introduced.
Expert Commentary:
Patisiran offers a new treatment option for patients with hereditary transthyretin-mediated amyloidosis. Patisiran can significantly reduce the TTR level and improve patient’s neuropathy and quality of life. The common adverse reactions were upper respiratory tract infections and infusion-related reactions.
organization: Central Hospital of Linyi City, ChinaDOI: 10.1080/17512433.2019.1567326
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