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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Vydáno v:Neurol Ther
Hlavní autoři: Urits, Ivan, Swanson, Daniel, Swett, Michael C., Patel, Anjana, Berardino, Kevin, Amgalan, Ariunzaya, Berger, Amnon A., Kassem, Hisham, Kaye, Alan D., Viswanath, Omar
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Healthcare 2020
Témata:
Review
On-line přístup: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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