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

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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Main Authors: 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
Format: Article
Language:English
Published: Thieme Revinter Publicações 2024-04-01
Series:Arquivos de Neuro-Psiquiatria
Subjects:
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 Access:http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1781463
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http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1781463

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