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hemophilia A |
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| The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Still, Selden admits to being "thrilled" with the results since this is the first gene therapy for hemophilia A in people. Syntonix is pursuing other early-stage programs, including a long-acting factor VIII program, with the potential to improve the treatment of hemophilia A. |
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