GS-14-ENVISION, a phase 3 study to evaluate efficacy and safety of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1, in acute hepatic porphyria patients

Authors :: Craig Penz
Delia D'Avola
Paula Aguilera
Yutaka Horie
D. Montgomery Bissell
Karl E. Anderson
Amy Chan
Pauline Harper
Herbert L. Bonkovsky
Sioban Keel
Jeeyoung Oh
Paolo Ventura
Gary Liu
Janneke G. Langendonk
Amy Simon
Jiaan-Der Wang
Samuel M. Silver
Gayle Ross
Jerzy Windyga
Charles J. Parker
Laurent Gouya
Manisha Balwani
Aneta Ivanova
Quinn Dinh
Peter Stewart
John D. Phillips
David C. Rees
Bruce Ritchie
Ulrich Stölzel
Eliane Sardh
Penelope E. Stein
Source :: Journal of Hepatology. 70:e81-e82
Publication Year :: 2019
Publisher :: Elsevier BV, 2019.

Subjects :: Acute hepatic porphyria
Hepatology
biology
business.industry
RNA interference
Aminolevulinic acid synthase
biology.protein
Medicine
Phases of clinical research
Pharmacology
business
Therapeutic targeting

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