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

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 therapi...

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Detalles Bibliográficos
Autores principales: Adams, David, Algalarrondo, Vincent, Polydefkis, Michael, Sarswat, Nitasha, Slama, Michel S., Nativi-Nicolau, Jose
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Position Statement
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8489116/
https://www.ncbi.nlm.nih.gov/pubmed/34602081
http://dx.doi.org/10.1186/s13023-021-01960-9

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