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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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