A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

Hereditary variant transthyretin amyloidosis (ATTRv) is a rare genetic defect that affects about 5000–10,000 people worldwide, causing amyloidosis secondary to misfolding of mutant transthyretin (TTR) protein fibrils. TTR mutations can cause protein deposits in many extracellular regions of organs,...

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Библиографические подробности
Опубликовано в: :Neurol Ther
Главные авторы: Urits, Ivan, Swanson, Daniel, Swett, Michael C., Patel, Anjana, Berardino, Kevin, Amgalan, Ariunzaya, Berger, Amnon A., Kassem, Hisham, Kaye, Alan D., Viswanath, Omar
Формат: Artigo
Язык:Inglês
Опубликовано: Springer Healthcare 2020
Предметы:
Review
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC7606409/
https://ncbi.nlm.nih.gov/pubmed/32785879
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40120-020-00208-1
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