Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression

Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression

Abstract Background Hereditary transthyretin-mediated amyloidosis, also known as ATTRv amyloidosis (v for variant), is a rare, autosomal dominant, fatal disease, in which systemic amyloid progressively impairs multiple organs, leading to disability and death. The recent approval of disease-modifying...

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Bibliographic Details
Main Authors: David Adams, Vincent Algalarrondo, Michael Polydefkis, Nitasha Sarswat, Michel S. Slama, Jose Nativi-Nicolau
Format: Article
Language:English
Published: BMC 2021-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
hATTR amyloidosis
ATTRv amyloidosis
Amyloid neuropathies
Amyloid cardiomyopathies
Familial
Diagnostic techniques and procedures
Online Access:https://doi.org/10.1186/s13023-021-01960-9

Internet

https://doi.org/10.1186/s13023-021-01960-9

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