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BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

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Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here.


BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A.
The data presented at ASH is the most current data (Nov. 16, 2017 cut off) and includes 78 weeks of data for the 6e13 vg/kg dose and 48 weeks of data for the 4e13 vg/kg dose.

The New England Journal of Medicine has published interim data.

 

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