Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B
作者
Steven W. Pipe,Wolfgang Miesbach,Michael Recht,Frank W.G. Leebeek,Nigel S. Key,Giancarlo Castaman,Susan Lattimore,Michiel Coppens,Sandra Le Quellec,Vaibhav Mahajan,Sean E. Gill,Douglass Drelich,Paul E. Monahan
Sustained endogenous factor IX expression and low annualized bleeding rates over a 5-year period were observed after an infusion of etranacogene dezaparvovec. (Funded by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.).
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(2025-6-4)
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