A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

Abstract Hereditary variant transthyretin amyloidosis (ATTRv) is a rare genetic defect that affects about 5000–10,000 people worldwide, causing amyloidosis secondary to misfolding of mutant transthyretin (TTR) protein fibrils. TTR mutations can cause protein deposits in many extracellular regions of...

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Bibliographic Details
Main Authors: Ivan Urits, Daniel Swanson, Michael C. Swett, Anjana Patel, Kevin Berardino, Ariunzaya Amgalan, Amnon A. Berger, Hisham Kassem, Alan D. Kaye, Omar Viswanath
Format: Article
Language:English
Published: Adis, Springer Healthcare 2020-08-01
Series:Neurology and Therapy
Subjects:
Cardiac amyloidosis
Hereditary amyloidosis
Orphan drugs
Polyneuropathy
Online Access:https://doi.org/10.1007/s40120-020-00208-1

Internet

https://doi.org/10.1007/s40120-020-00208-1

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