Emerging therapies in transthyretin amyloidosis - a new wave of hope after years of stagnancy?

Abstract:

:Transthyretin amyloidosis (ATTR) is a rare, yet underdiagnosed disease characterized by progressive impairment of neurologic and cardiac function due to deposition of misfolded transthyretin. Despite great efforts, such as the introduction of orthotopic liver transplant, the devastating prognosis for both variant and wild-type ATTR patients remained unchanged over the last decades, mainly due to a lack of specific therapies. Fortunately, recent years saw the introduction of promising targeted therapies, which aim to interfere with the deposition of misfolded transthyretin (TTR) at various stages of the cascade underlying ATTR progression. These include TTR tetramer stabilizers (tafamidis, diflunisal, epigallocatechin-3-gallate), TTR silencers (inotersen, patisiran) and fibril disruptors (monoclonal antibodies, doxycycline and tauroursodeoxycholic acid). In the context of this review we explain their mechanisms of action, analyse their efficacy on neurologic and cardiac function based on all clinical trials conducted to date and discuss their clinical applicability. Eventually suggestions for future clinical research into the field are provided.

journal_name

Eur J Heart Fail

authors

Müller ML,Butler J,Heidecker B

doi

10.1002/ejhf.1695

subject

Has Abstract

pub_date

2020-01-01 00:00:00

pages

39-53

issue

1

eissn

1388-9842

issn

1879-0844

journal_volume

22

pub_type

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