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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Detalhes bibliográficos
Publicado no:Neurol Ther
Main Authors: Urits, Ivan, Swanson, Daniel, Swett, Michael C., Patel, Anjana, Berardino, Kevin, Amgalan, Ariunzaya, Berger, Amnon A., Kassem, Hisham, Kaye, Alan D., Viswanath, Omar
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Healthcare 2020
Assuntos:
Review
Acesso em linha: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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