BioMarin to Begin Phase 3 Gene Therapy Trials for Hemophilia A – Rare Disease Report
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BioMarin to Begin Phase 3 Gene Therapy Trials for Hemophilia A
Rare Disease Report Hemophilia A is a chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of Factor VIII. People with this rare disorder experience recurrent painful bleeding episodes, some of which can be life-threatening. BioMarin Announces Plans to Progress Both the 6e13vg/kg and 4e13 vg/kg Doses of BMN 270, its Investigational … |
