Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

2018 New England Journal of Medicine 2,680 citations

Abstract

In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis. (Funded by Alnylam Pharmaceuticals; APOLLO ClinicalTrials.gov number, NCT01960348 .).

Keywords

TransthyretinRNA interferenceAmyloidosisMedicineCancer researchRNAGeneticsBiologyInternal medicineGene

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Publication Info

Year
2018
Type
article
Volume
379
Issue
1
Pages
11-21
Citations
2680
Access
Closed

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David Adams, Alejandra González‐Duarte, William O’Riordan et al. (2018). Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. New England Journal of Medicine , 379 (1) , 11-21. https://doi.org/10.1056/nejmoa1716153

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DOI
10.1056/nejmoa1716153