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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Detaylı Bibliyografya
Yayımlandı:Brain Sci
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: MDPI 2021
Konular:
Article
Online Erişim: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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