Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial

Margareth C. Ozelo, MD, PhD

Margareth C. Ozelo, MD, PhD

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

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