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BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A

Published: 2022-03-17 12:31:00 ET
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New England Journal of Medicine Publishes Editorial: Prepare the Way for Hemophilia A Gene Therapy

Significantly Reduced Bleeding and Factor VIII Utilization Compared to Baseline Factor VIII Prophylactic Therapy

Mean Annualized Bleeding Rate (ABR) Reduced by 84% (p-value