Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis.

Patisiran for the treatment of hereditary transthyretin-mediated amyloidosis. Expert Rev Clin Pharmacol. 2019 Jan 15;: Authors: Yang J Abstract INTRODUCTION: 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, 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. PMID: 30644768 [PubMed - as supplied by publisher]
Source: Expert Review of Clinical Pharmacology - Category: Drugs & Pharmacology Tags: Expert Rev Clin Pharmacol Source Type: research