BioMarin Provides 3 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

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May 28, 2019
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Substantial Reduction in excess of 92% in Mean Bleed Rate Requiring Factor VIII Infusions; Sustained over the Period of Observation (3 Years in High Dose and 2 Years in Low Dose)

Rate of FVIII Change Continued to be Expression Level Dependent, Slowed in Year 3, and Appears to be Approaching Plateau

Conference Call and Webcast to be Held Tuesday, May 28, 2019 at 8:00 AM Eastern

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