Real-life experience with inotersen at CEPARM, Hospital Universitário Clementino Fraga Filho, Universidade Federal do Rio de Janeiro

Background Hereditary transthyretin amyloidosis (ATTRv) is an inherited, progressive, and fatal disease still largely underdiagnosed. Mutations in the transthyretin (TTR) gene cause the TTR protein to destabilize, misfold, aggregate, and deposit in body tissues, which makes ATTRv a diseas...

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Bibliografische gegevens

Hoofdauteurs:	Moises Dias, Luiz Felipe Pinto, Marcus Vinícius Pinto, Renata Gervais, Paula Accioli, Gabriela Amorim, Mariana Guedes, Carlos Perez Gomes, Roberto Coury Pedrosa, Márcia Waddington-Cruz
Formaat:	Artigo
Taal:	Inglês
Gepubliceerd in:	Thieme Revinter Publicações 2024-04-01
Reeks:	Arquivos de Neuro-Psiquiatria
Onderwerpen:	Amyloidosis Peripheral Nervous System Diseases Amyloid Neuropathies, Familial Mutation, Missense Amyloidosis, Hereditary, Transthyretin-Related Inotersen Amiloidose Doenças do Sistema Nervoso Periférico Neuropatias Amiloides Familiares Mutação de Sentido Incorreto Amiloidose Neuropática Hereditária Inotersena
Online toegang:	http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1781463
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