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Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147.
N Engl J Med. 2020.
PMID: 32521132
Clinical Trial.
Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial.
Kuter DJ, Bonkovsky HL, Monroy S, Ross G, Guillén-Navarro E, Cappellini MD, Minder AE, Hother-Nielsen O, Ventura P, Jia G, Sweetser MT, Thapar M; ENVISION Investigators.
Kuter DJ, et al.
J Hepatol. 2023 Nov;79(5):1150-1158. doi: 10.1016/j.jhep.2023.06.013. Epub 2023 Jul 20.
J Hepatol. 2023.
PMID: 37479139
Free article.
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Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study.
Ventura P, Bonkovsky HL, Gouya L, Aguilera-Peiró P, Montgomery Bissell D, Stein PE, Balwani M, Anderson DKE, Parker C, Kuter DJ, Monroy S, Oh J, Ritchie B, Ko JJ, Hua Z, Sweetser MT, Sardh E; ENVISION Investigators.
Ventura P, et al.
Liver Int. 2022 Jan;42(1):161-172. doi: 10.1111/liv.15090. Epub 2021 Nov 16.
Liver Int. 2022.
PMID: 34717041
Free PMC article.
Clinical Trial.
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