Patisiran in hATTR Amyloidosis: Six-Month Latency Period before Efficacy

Hereditary amyloidosis associated with mutations in the transthyretin gene (hATTR) is a progressive devastating disease, with a fatal outcome occurring within 10years after onset. In recent years, TTR gene silencing therapy appeared as a promising therapeutic strategy, showing evidence that disease...

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Detalhes bibliográficos
Publicado no:Brain Sci
Main Authors: Gentile, Luca, Russo, Massimo, Luigetti, Marco, Bisogni, Giulia, Di Paolantonio, Andrea, Romano, Angela, Guglielmino, Valeria, Arimatea, Ilenia, Sabatelli, Mario, Toscano, Antonio, Vita, Giuseppe, Mazzeo, Anna
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2021
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8073153/
https://ncbi.nlm.nih.gov/pubmed/33921571
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/brainsci11040515
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