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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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Bibliografski detalji
Izdano u:Neurol Ther
Glavni autori: Urits, Ivan, Swanson, Daniel, Swett, Michael C., Patel, Anjana, Berardino, Kevin, Amgalan, Ariunzaya, Berger, Amnon A., Kassem, Hisham, Kaye, Alan D., Viswanath, Omar
Format: Artigo
Jezik:Inglês
Izdano: Springer Healthcare 2020
Teme:
Online pristup: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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