Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-state Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderately Severe Hemophilia B
Highlights From the 15th Annual Congress of EAHAD

Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-state Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderately Severe Hemophilia B

Wolfgang Miesbach1; Frank W.G.Leebeek2; Michael Recht3; Nigel S. Key4; Susan Lattimore3; Giancarlo Castaman5; Michiel Coppens6; David Cooper7; Sergio Slawka7; Stephanie Verweij7; Robert Gut7; Ricardo Dolmetsch7; Yanyan Li8; Paul E. Monahan8; Steven W. Pipe9

HOPE-B Investigators1; University Hospital Frankfurt, Frankfurt, Germany2; Erasmus MC, University Medical Center Rotterdam, Netherlands3; Oregon Health & Science University, Portland, OR, United States4; University of North Carolina, Chapel Hill, NC, United States5; Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy6; Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands7; uniQureBV, Amsterdam, Netherlands/uniQure Inc. Lexington, MA, USA8; CSL Behring, King of Prussia, PA, UnitedStates9; University of Michigan, Ann Arbor, MI, United States

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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