Gene therapy trials in lab animals show significant increases in production of factor VIII and IX.
Early clinical trial results in patients with hemophilia A show increased clotting factor levels in all subjects.
New agent will provide another option for promoting blood clotting in patients with bleeding disorders undergoing dental procedures.
The collaboration will focus on developing adeno-associated viruses (AAVs), delivery vehicles for carrying genetic material, including those for hemophilia.
Alnylam Pharmaceuticals announces Phase 1 study of ALN-AT3, a subcutaneous therapy being tested on patients with hemophilia.
BioMarin announces enrollment of the first patient in clinical trial of BMN 270, a gene therapy to treat people with hemophilia A.
Genentech receives breakthrough therapy designation from the FDA for ACE910 subcutaneous therapy for hemophilia A.
IXINITY® provides good bleed control for patients with severe to moderately severe hemophilia B.
New recombinant FVIII therapy reduces bleeding frequency and offers a new option for patients.
Pharmaceutical company donates 2 million international units of protein factor therapies to the World Federation of Hemophilia (WFH).
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