Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

Balwani, Manisha; Sardh, Eliane; Ventura, Paolo; Aguilera Peiró, Paula; C. Rees, David; Stölzel, Ulrich; Montgomery Bissell, D.; L. Bonkovsky, Herbert; Windyga, Jerzy; E. Anderson, Karl; Parker, Charles; Silver, Samuel; Keel, Sioḃán; Wang, Jiaan‐Der; E. Stein, Penelope; Harper, Pauline; Vassiliou, Daphne; Wang, Bruce; D. Phillips, John; Ivanova, Aneta; G. Langendonk, Janneke; Kauppinen, Raili; I. Minder, Elisabeth; Horie, Yutaka; Penz, Craig; Chen, Jihong; Liu, Shangbin; J. Ko, John; T. Sweetser, Marianne; Garg, Pushkal; Vaishnaw, Akshay; Bum Kim, Jae; Simon, Amy; Gouya, Laurent

doi:10.1056/nejmoa1913147

Public

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

Published in New England Journal of Medicine • Jun 10, 2020

Authors:

Manisha Balwani

Eliane Sardh

Paolo Ventura

Abstract

BACKGROUND: Up-regulation of hepatic delta-aminolevulinic acid synthase 1 (ALAS1), with resultant accumulation of delta-aminolevulinic acid (ALA) and porphobilinogen, is central to the pathogenesis of acute attacks and chronic symptoms in acute hepatic porphyria. Givosiran, an RNA interference thera...

Subject

RNA interference

Acute intermittent porphyria

Porphyria

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