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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Dades bibliogràfiques
Publicat a:Neurol Ther
Autors principals: 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
Idioma:Inglês
Publicat: Springer Healthcare 2020
Matèries:
Review
Accés en línia: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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