Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

M Balwani; E Sardh; P Ventura; PA Peiro; DC Rees; U Stolzel; DM Bissell; HL Bonkovsky; J Windyga; KE Anderson; C Parker; SM Silver; SB Keel; JD Wang; PE Stein; P Harper; D Vassiliou; B Wang; J Phillips; A Ivanova; Janneke Langendonk; R Kauppinen; E Minder; Y Horie; C Penz; JH Chen; SB Liu; JJ Ko; MT Sweetser; P Garg; A Vaishnaw; JB Kim; AR Simon; L Gouya

doi:10.1056/NEJMoa1913147

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

M Balwani, E Sardh, P Ventura, PA Peiro, DC Rees, U Stolzel, DM Bissell, HL Bonkovsky, J Windyga, KE Anderson, C Parker, SM Silver, SB Keel, JD Wang, PE Stein, P Harper, D Vassiliou, B Wang, J Phillips, A IvanovaJanneke Langendonk, R Kauppinen, E Minder, Y Horie, C Penz, JH Chen, SB Liu, JJ Ko, MT Sweetser, P Garg, A Vaishnaw, JB Kim, AR Simon, L Gouya

Internal Medicine

Research output: Contribution to journal › Article › Academic › peer-review

218 Citations (Scopus)

Original language	English
Pages (from-to)	2289-2301
Number of pages	13
Journal	New England Journal of Medicine
Volume	382
Issue number	24
DOIs	https://doi.org/10.1056/NEJMoa1913147
Publication status	Published - 2020

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10.1056/NEJMoa1913147

http://hdl.handle.net/1765/128419

Cite this

Balwani, M., Sardh, E., Ventura, P., Peiro, PA., Rees, DC., Stolzel, 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., ... Gouya, L. (2020). Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. New England Journal of Medicine, 382(24), 2289-2301. https://doi.org/10.1056/NEJMoa1913147

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title = "Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria",

author = "M Balwani and E Sardh and P Ventura and PA Peiro and DC Rees and U Stolzel and DM Bissell and HL Bonkovsky and J Windyga and KE Anderson and C Parker and SM Silver and SB Keel and JD Wang and PE Stein and P Harper and D Vassiliou and B Wang and J Phillips and A Ivanova and Janneke Langendonk and R Kauppinen and E Minder and Y Horie and C Penz and JH Chen and SB Liu and JJ Ko and MT Sweetser and P Garg and A Vaishnaw and JB Kim and AR Simon and L Gouya",

year = "2020",

doi = "10.1056/NEJMoa1913147",

language = "English",

volume = "382",

pages = "2289--2301",

journal = "New England Journal of Medicine",

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Balwani, M, Sardh, E, Ventura, P, Peiro, PA, Rees, DC, Stolzel, 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, J, Kauppinen, R, Minder, E, Horie, Y, Penz, C, Chen, JH, Liu, SB, Ko, JJ, Sweetser, MT, Garg, P, Vaishnaw, A, Kim, JB, Simon, AR & Gouya, L 2020, 'Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria', New England Journal of Medicine, vol. 382, no. 24, pp. 2289-2301. https://doi.org/10.1056/NEJMoa1913147

TY - JOUR

T1 - Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

AU - Balwani, M

AU - Sardh, E

AU - Ventura, P

AU - Peiro, PA

AU - Rees, DC

AU - Stolzel, U

AU - Bissell, DM

AU - Bonkovsky, HL

AU - Windyga, J

AU - Anderson, KE

AU - Parker, C

AU - Silver, SM

AU - Keel, SB

AU - Wang, JD

AU - Stein, PE

AU - Harper, P

AU - Vassiliou, D

AU - Wang, B

AU - Phillips, J

AU - Ivanova, A

AU - Langendonk, Janneke

AU - Kauppinen, R

AU - Minder, E

AU - Horie, Y

AU - Penz, C

AU - Chen, JH

AU - Liu, SB

AU - Ko, JJ

AU - Sweetser, MT

AU - Garg, P

AU - Vaishnaw, A

AU - Kim, JB

AU - Simon, AR

AU - Gouya, L

PY - 2020

Y1 - 2020

U2 - 10.1056/NEJMoa1913147

DO - 10.1056/NEJMoa1913147

M3 - Article

VL - 382

SP - 2289

EP - 2301

JO - New England Journal of Medicine

JF - New England Journal of Medicine

SN - 0028-4793

IS - 24

ER -