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EIGHTEEN-MONTH INTERIM ANALYSIS OF EFFICACY AND SAFETY OF GIVOSIRAN, AN RNAI THERAPEUTIC FOR ACUTE HEPATIC PORPHYRIA, IN THE ENVISION OPEN LABEL EXTENSION
D Kutera, S Keelb, C Parkerc, DC Reesd, U Stölzele, P Venturaf, M Balwanig, L Gouyah, S Rhyeei, S Silverj
a Massachusetts General Hospital, Boston, United States
b University of Washington, Seattle, United States
c University of Utah, Salt Lake City, United States
d King's College Hospital, London, United Kingdom
e Klinikum Chemnitz, Chemnitz, Germany
f Università degli Studi di Modena e Reggio Emilia, Modena, Italy
g Icahn School of Medicine at Mt. Sinai, New York, United States
h Centre Français des Porphyries, Paris, France
i Alnylam Pharmaceuticals, Cambridge, United States
j University of Michigan, Ann Arbor, United States
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ISSN: 25311379
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Idiomas
Hematology, Transfusion and Cell Therapy