机译:Efficacy and safety of givosiran for acute hepatic porphyria: 24‐month interim analysis of the randomized phase 3 ENVISION study
Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit;
Section on Gastroenterology and Hepatology,Wake Forest University/North Carolina Baptist Medical;
Centre Fran?ais des PorphyriesHospital Clinic BarcelonaUCSF Liver Center and Porphyria Center, University of CaliforniaKing’s College HospitalDepartment of Genetics and Genomic Sciences, Icahn School of Medicine at Mount SinaiUniversity of Texas Medical BranchUniversity of UtahCenter for Hematology,Massachusetts General HospitalInstituto Nacional de PediatríaKonkuk University Medical CenterUniversity of Alberta HospitalAlnylam PharmaceuticalsPorphyria Centre Sweden, Centre for Inherited Metabolic Diseases, Karolinska Institutet, Karolinska;
Acute hepatic porphyria; ALA synthase‐1; givosiran; health‐related quality of life; RNAi therapeutics;