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TWELVE-MONTH INTERIM ANALYSIS OF EFFICACY AND SAFETY OF GIVOSIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC FOR ACUTE HEPATIC PORPHYRIA, IN THE ENVISION OPEN LABEL EXTENSION
R. Marchia, L. Duartea, S. Eb, B. Mc, R. Dcd, S. Pd, S. Ue, A.P. Pf, B. Dmg, E. Ala, K. Sh, P. Ci, P. Jdi
a Alnylam Brasil Farmacêutica Ltda, São Paulo, SP, Brazil
b Porphyria Centre Sweden, Centre for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden
c Icahn School of Medicine at Mt. Sinai, New York, United States
d King's College Hospital, London, United Kingdom
e Klinikum Chemnitz, Chemnitz, Germany
f Hospital Clinic Barcelona, Barcelona, Spain
g University of California, San Francisco, United States
h University of Washington, Seattle, United States
i University of Utah, Salt Lake City, United States
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| 2024 April | 12 | 9 | 21 |
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| 2023 December | 17 | 18 | 35 |
| 2023 November | 34 | 30 | 64 |
| 2023 October | 15 | 27 | 42 |
| 2023 September | 21 | 28 | 49 |
| 2023 August | 13 | 12 | 25 |
| 2023 July | 18 | 14 | 32 |
| 2023 June | 22 | 17 | 39 |
| 2023 May | 25 | 13 | 38 |
| 2023 April | 19 | 15 | 34 |
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| 2023 January | 13 | 16 | 29 |
| 2022 December | 25 | 18 | 43 |
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| 2022 August | 13 | 20 | 33 |
| 2022 July | 19 | 26 | 45 |
| 2022 June | 18 | 19 | 37 |
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| 2022 April | 18 | 29 | 47 |
| 2022 March | 16 | 22 | 38 |
| 2022 February | 15 | 22 | 37 |
| 2022 January | 12 | 14 | 26 |
| 2021 December | 14 | 20 | 34 |
| 2021 November | 23 | 26 | 49 |
| 2021 October | 35 | 35 | 70 |
| 2021 September | 27 | 27 | 54 |
| 2021 August | 24 | 40 | 64 |
| 2021 July | 25 | 25 | 50 |
| 2021 June | 16 | 22 | 38 |
| 2021 May | 22 | 58 | 80 |
| 2021 April | 49 | 60 | 109 |
| 2021 March | 29 | 30 | 59 |
| 2021 February | 19 | 19 | 38 |
| 2021 January | 21 | 27 | 48 |
| 2020 December | 26 | 22 | 48 |
| 2020 November | 28 | 19 | 47 |
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