Disease-Modifying Treatments for Transthyretin Amyloidosis.
J Cardiovasc Pharmacol
; 78(5): e641-e647, 2021 11 01.
Article
em En
| MEDLINE
| ID: mdl-34321398
ABSTRACT
ABSTRACT The transthyretin (TTR) amyloidoses result from misfolding of the protein leading to fibril formation and aggregation as amyloid deposits in predominantly the cardiovascular and nervous systems. Cardiac involvement can manifest as heart failure, arrhythmias, and valvular disease. Neurologic involvement can cause sensorimotor polyneuropathies, mononeuropathies, and dysautonomia. Previously, treatment has focused on management of these symptoms and disease sequelae, with a high rate of mortality due to the absence of disease-modifying therapies. In this article, we review novel treatments focusing on 3 mechanistic pathways (1) silencing of the TTR gene to suppress production, (2) stabilizing of TTR tetramers to prevent misfolding, or (3) disrupting of existing TTR amyloid fibrils to promote reabsorption.
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Base de dados:
MEDLINE
Assunto principal:
Pré-Albumina
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Fármacos Cardiovasculares
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Terapia Genética
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Neuropatias Amiloides Familiares
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Miócitos Cardíacos
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Amiloide
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Cardiomiopatias
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article