Emily Symington

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A

John Pasi, Savita Rangarajan, Nina Mitchell et al. · New England Journal of Medicine · 2020 · 441 citations · Full text

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht et al. · New England Journal of Medicine · 2023 · 307 citations · Full text

Diagnostic high-throughput sequencing of 2396 patients with bleeding, thrombotic, and platelet disorders

Kate Downes, Karyn Mégy, Daniel Duarte et al. · Blood · 2019 · 210 citations · Full text

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Johnny Mahlangu, Radosław Kaczmarek, Annette von Drygalski et al. · New England Journal of Medicine · 2023 · 176 citations · Full text

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

John Pasi, Michael Laffan, Savita Rangarajan et al. · Haemophilia · 2021 · 95 citations · Full text