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Journal Abstract Search

285 related items for PubMed ID: 35067977

  • 1.
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  • 2. 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.
    N Engl J Med; 2020 Jun 11; 382(24):2289-2301. PubMed ID: 32521132
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  • 3. Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
    Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE.
    N Engl J Med; 2019 Feb 07; 380(6):549-558. PubMed ID: 30726693
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  • 5. Givosiran: A Review in Acute Hepatic Porphyria.
    Syed YY.
    Drugs; 2021 May 07; 81(7):841-848. PubMed ID: 33871817
    [Abstract] [Full Text] [Related]

  • 6. Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.
    Wang B, Ventura P, Takase KI, Thapar M, Cassiman D, Kubisch I, Liu S, Sweetser MT, Balwani M.
    Orphanet J Rare Dis; 2022 Aug 26; 17(1):327. PubMed ID: 36028858
    [Abstract] [Full Text] [Related]

  • 7. 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.
    Liver Int; 2022 Jan 26; 42(1):161-172. PubMed ID: 34717041
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  • 9. Givosiran to treat acute porphyria.
    Honor A, Rudnick SR, Bonkovsky HL.
    Drugs Today (Barc); 2021 Jan 26; 57(1):47-59. PubMed ID: 33594389
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  • 12. Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study.
    Sardh E, Balwani M, Rees DC, Anderson KE, Jia G, Sweetser MT, Wang B.
    Orphanet J Rare Dis; 2024 Oct 03; 19(1):365. PubMed ID: 39363243
    [Abstract] [Full Text] [Related]

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  • 14. RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice.
    Yasuda M, Gan L, Chen B, Kadirvel S, Yu C, Phillips JD, New MI, Liebow A, Fitzgerald K, Querbes W, Desnick RJ.
    Proc Natl Acad Sci U S A; 2014 May 27; 111(21):7777-82. PubMed ID: 24821812
    [Abstract] [Full Text] [Related]

  • 15. 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.
    J Hepatol; 2023 Nov 27; 79(5):1150-1158. PubMed ID: 37479139
    [Abstract] [Full Text] [Related]

  • 16. RNA interference therapy in acute hepatic porphyrias.
    Yasuda M, Keel S, Balwani M.
    Blood; 2023 Nov 09; 142(19):1589-1599. PubMed ID: 37027823
    [Abstract] [Full Text] [Related]

  • 17.
    Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, Sardh E, Barbaro M.
    ; 1993 Nov 09. PubMed ID: 20301372
    [Abstract] [Full Text] [Related]

  • 18. Novel treatment options for acute hepatic porphyrias.
    Wang B.
    Curr Opin Gastroenterol; 2021 May 01; 37(3):194-199. PubMed ID: 33769375
    [Abstract] [Full Text] [Related]

  • 19. 5-Aminolevulinate dehydratase porphyria: Update on hepatic 5-aminolevulinic acid synthase induction and long-term response to hemin.
    Lahiji AP, Anderson KE, Chan A, Simon A, Desnick RJ, Ramanujam VMS.
    Mol Genet Metab; 2020 Dec 01; 131(4):418-423. PubMed ID: 33199206
    [Abstract] [Full Text] [Related]

  • 20. A Drug-Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver.
    Vassiliou D, Sardh E, Harper P, Simon AR, Clausen VA, Najafian N, Robbie GJ, Agarwal S.
    Clin Pharmacol Ther; 2021 Nov 01; 110(5):1250-1260. PubMed ID: 34510420
    [Abstract] [Full Text] [Related]

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