ASH17: BioMarin’s gene therapy promises one-time treatment of hemophilia – BioPharma Dive
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ASH17: BioMarin's gene therapy promises one-time treatment of hemophilia
BioPharma Dive An experimental gene therapy from BioMarin Pharmaceuticals Inc. dramatically reduced bleeding episodes in patients with hemophilia A, boosting levels of the blood clotting protein Factor VIII to near-normal or normal levels in 11 out of 13 patients … |
