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BioMarin Announces Durable Hemostatic Efficacy Maintained Over 6 years in Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A

Published: May 31, 2022

95% Reduction in Mean Annualized Bleed Rate (ABR) and 96% Reduction in Mean Annualized Factor VIII Usage from Baseline Through Year 6 in 6e13 vg/kg Dose Cohort...

Source: prnewswire.com

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United StatesCity OfUnited KingdomHank FuchsDebra CharlesworthMichael LaffanTraci MccartyHammersmith Hospital Haemophilia CentreInternational Society On ThrombosisEuropean CommissionDrug AdministrationBiomarin Pharmaceutical IncData Monitoring CommitteeWorldwide ResearchExchange CommissionImperial College LondonEuropean Medicines AgencyDepartment Of ImmunologyCommittee For Medicinal Products Human UseMean Annualized Bleed RateMean Annualized FactorBaseline Through YearOral PresentationUpcoming International SocietySnow Expected