Disease-Modifying Drugs Extend Survival in Hereditary Transthyretin Amyloid Polyneuropathy.
Ann Neurol
; 95(2): 230-236, 2024 Feb.
Article
em En
| MEDLINE
| ID: mdl-38053464
ABSTRACT
Hereditary transthyretin (ATTRv) amyloidosis is a rare, fatal systemic disease, associated with polyneuropathy and cardiomyopathy, that is caused by mutant transthyretin (TTR). In addition to liver transplantation, several groundbreaking disease-modifying drugs (DMDs) such as tetrameric TTR stabilizers and TTR gene-silencing therapies have been developed for ATTRv amyloid polyneuropathy. They were based on a working hypothesis of the mechanisms of ATTRv amyloid formation. In this retrospective cohort study, we investigated survival of all 201 consecutive patients with ATTRv amyloidosis in our center. The effects of DMDs on survival improvements were significant not only in early-onset patients but also in late-onset patients. ANN NEUROL 2024;95230-236.
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1
Base de dados:
MEDLINE
Assunto principal:
Polineuropatias
/
Neuropatias Amiloides
/
Neuropatias Amiloides Familiares
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article