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Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Balwani M, Sardh E, Ventura P, Peiró PA, Rees DC, Stölzel U, Bissell DM, Bonkovsky HL, Windyga J, Anderson KE, Parker C, Silver SM, Keel SB, Wang JD, Stein PE, Harper P, Vassiliou D, Wang B, Phillips J, Ivanova A, Langendonk JG, Kauppinen R, Minder E, Horie Y, Penz C, Chen J, Liu S, Ko JJ, Sweetser MT, Garg P, Vaishnaw A, Kim JB, Simon AR, Gouya L; ENVISION Investigators. Balwani M, et al. Among authors: ko jj. N Engl J Med. 2020 Jun 11;382(24):2289-2301. doi: 10.1056/NEJMoa1913147. N Engl J Med. 2020. PMID: 32521132 Clinical Trial.
EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.
Gouya L, Ventura P, Balwani M, Bissell DM, Rees DC, Stölzel U, Phillips JD, Kauppinen R, Langendonk JG, Desnick RJ, Deybach JC, Bonkovsky HL, Parker C, Naik H, Badminton M, Stein PE, Minder E, Windyga J, Bruha R, Cappellini MD, Sardh E, Harper P, Sandberg S, Aarsand AK, Andersen J, Alegre F, Ivanova A, Talbi N, Chan A, Querbes W, Ko J, Penz C, Liu S, Lin T, Simon A, Anderson KE. Gouya L, et al. Hepatology. 2020 May;71(5):1546-1558. doi: 10.1002/hep.30936. Epub 2019 Nov 7. Hepatology. 2020. PMID: 31512765 Free PMC article.
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. Among authors: ko jj. 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.
Quantifying the impact of symptomatic acute hepatic porphyria on well-being via patient-reported outcomes: Results from the Porphyria Worldwide Patient Experience Research (POWER) study.
Dickey A, Wheeden K, Lyon D, Burrell S, Hegarty S, Falchetto R, Williams ER, Barman-Aksözen J, DeCongelio M, Bulkley A, Matos JE, Mnif T, Mora J, Ko JJ, Meninger S, Lombardelli S, Nance D. Dickey A, et al. Among authors: ko jj. JIMD Rep. 2022 Oct 18;64(1):104-113. doi: 10.1002/jmd2.12343. eCollection 2023 Jan. JIMD Rep. 2022. PMID: 36636593 Free PMC article.
172 results