Patisiran for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis | oneAMYLOIDOSISvoice
Scientific Articles

Patisiran for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis

key information

source: Expert Review of Clinical Pharmacology

year: 2019

authors: Yang J


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, China

DOI: 10.1080/17512433.2019.1567326

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