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BioMarin Announces New England Journal of Medicine Publishes 3 Years of Follow-up Data in Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Single Dose Resulted in Sustained Mean Reduction of 96% in Bleed Rates at 6e13 vg/kg Dose in Third YearCompletely Eliminated Prophylactic Factor VIII Use in 6e13 vg/kg and 4e13 vg/kg Doses2nd Publication in NEJM on Valoctocogene Roxaparvovec

SAN RAF… […]