Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

20182,836 citationsJournal Articlebronze Open Access

Authors

David Adams · Inserm

Alejandra González‐Duarte · Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán

William O’Riordan

Chih‐Chao Yang · National Taiwan University Hospital

Mitsuharu Ueda

Arnt V. Kristen · Heidelberg University

Ivailo Tournev · National Institute of Emergency Medicine "Pirogov"

Hartmut Schmidt

Abstract

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

Topics & Keywords

Amyloidosis: Diagnosis, Treatment, Outcomes Peptidase Inhibition and Analysis Eosinophilic Disorders and Syndromes

Publication Details

Published in: New England Journal of Medicine

Volume 379, Issue 1, pp. 11-21

DOI: 10.1056/nejmoa1716153

Field-Weighted Citation Impact: 106.91

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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

Authors

Abstract

Topics & Keywords

Publication Details