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FDA Grants Orphan Drug Designation to Biomarin's BMN-270 for Hemophilia A

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Additional Information

Additional Relevant Details BMN 270 is an AAV 5 factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A.   BMN-270 is currently in a phase 1/2 study of up to 12 patients to test efficacy and safety of BMN-270.
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Occurred on:
Mar 01, 2016
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Related Keywords Orphan Drug Designation, Bmn-270, Fda, Hemophilia A