Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Author:

Mahlangu Johnny1,Kaczmarek Radoslaw1,von Drygalski Annette1,Shapiro Susan1,Chou Sheng-Chieh1,Ozelo Margareth C.1ORCID,Kenet Gili1,Peyvandi Flora1ORCID,Wang Michael1,Madan Bella1,Key Nigel S.1,Laffan Michael1,Dunn Amy L.1,Mason Jane1,Quon Doris V.1,Symington Emily1,Leavitt Andrew D.1,Oldenburg Johannes1,Chambost Hervé1,Reding Mark T.1,Jayaram Kala1,Yu Hua1,Mahajan Reena1,Chavele Konstantia-Maria1,Reddy Divya B.1,Henshaw Joshua1,Robinson Tara M.1,Wong Wing Yen1,Pipe Steven W.1

Affiliation:

1. From the Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand, and National Health Laboratory Service, Johannesburg (J. Mahlangu); the Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis (R.K.); the Laboratory of Glycobiology, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland (R.K.); the Department of Medicine, University of California, San Diego...

Funder

BioMarin Pharmaceutical

Publisher

Massachusetts Medical Society

Subject

General Medicine

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