Advances in the treatment of hereditary transthyretin amyloidosis: A review.

ABSTRACT:

Introduction

Amyloid transthyretin amyloidosis (ATTR) is a progressive and often fatal disease caused by the buildup of mutated (hereditary ATTR [hATTR]; also known as ATTR variant [ATTRv]) or normal transthyretin (wild-type ATTR) throughout the body. Two new therapies-inotersen, an antisense oligonucleotide therapy, and patisiran, an RNA interference therapy-received marketing authorization and represent a significant advance in the treatment of amyloidosis. Herein, we describe the clinical presentation of ATTR, commonly used procedures in its diagnosis, and current treatment landscape for ATTR, with a focus on hATTR.

Methods

A PubMed search from 2008 to September 2018 was conducted to review the literature on ATTR.

Results

Until recently, there have been few treatme

SUBMITTER: Gertz MA 

PROVIDER: S-EPMC6749475 | biostudies-literature | 2019 Sep

REPOSITORIES: biostudies-literature