Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial.

Kuter, David J; Bonkovsky, Herbert L; Monroy, Susana; Ross, Gayle; Guillén-Navarro, Encarna; Cappellini, Maria Domenica; Minder, Anna-Elisabeth; Hother-Nielsen, Ole; Ventura, Paolo; Jia, Gang; Sweetser, Marianne T; Thapar, Manish.

Afiliação

Kuter DJ; Hematology Division, Massachusetts General Hospital, Boston, MA, USA. Electronic address: dkuter@mgh.harvard.edu.
Bonkovsky HL; Atrium Wake Forest Baptist Health, Winston-Salem, NC, USA.
Monroy S; Instituto Nacional de Pediatría, Mexico City, Mexico.
Ross G; Royal Melbourne Hospital, Melbourne, Victoria, Australia.
Guillén-Navarro E; Medical Genetics Section, Virgen de la Arrixaca University Hospital, IMIB Pascual Parrilla, University of Murcia (UMU), Murcia, Spain; CIBERER-ISCIII, Madrid, Spain.
Cappellini MD; University of Milan, Fondazione IRCCS Ca' Granda Policlinico, Milan, Italy.
Minder AE; Division of Endocrinology, Diabetes and Porphyria, Stadtspital Zürich, Triemli, Zürich, Switzerland.
Hother-Nielsen O; Odense University Hospital, Odense, Denmark.
Ventura P; Department of Surgical and Medical Sciences for Children and Adults, Internal Medicine Unit, University of Modena and Reggio Emilia, Modena, Italy.
Jia G; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Sweetser MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
Thapar M; Thomas Jefferson University, Philadelphia, PA, USA.