Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Top Cited Papers
Open Access
- 5 July 2018
- journal article
- research article
- Published by Massachusetts Medical Society in The New England Journal of Medicine
- Vol. 379 (1), 11-21
- https://doi.org/10.1056/nejmoa1716153
Abstract
Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin.Keywords
Funding Information
- Alnylam Pharmaceuticals (Not applicable)
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