Press Releases
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Jul 11, 2022
Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, Ongoing Clinical Development Program for any Gene Therapy in Hemophilia A European Commission...
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Jun 24, 2022
European Commission Approval Expected Q3 2022 1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe More than 20,000 Adults with Severe Hemophilia A in BioMarin...
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Jun 21, 2022
No Lower Age Restriction for Treatment Japan Accounts for Approximately Half of the 1,500 Patient Opportunity in APAC Region SAN RAFAEL, Calif., June 21, 2022 /PRNewswire/ -- BioMarin...
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Jun 13, 2022
Improvement in Height Z-Score and Annualized Growth Velocity Observed, Consistent with Children Over 5 Interactions with Health Authorities Planned for H2 2022 to Discuss Expanding Access to...
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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 91% Reduction in Mean ABR and 93%...