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Publication | Open Access

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B

DOIFull Paper Access

307

Citations

28

References

2023

Year

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht, Nigel S. Key, Giancarlo Castaman, Wolfgang Miesbach, Susan Lattimore, Kathelijne Peerlinck, Paul van der Valk, Michiel Coppens,
New England Journal of Medicine

Abstract

Etranacogene dezaparvovec gene therapy was superior to prophylactic factor IX with respect to the annualized bleeding rate, and it had a favorable safety profile. (Funded by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.).

References

YearCitations

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