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By BioMarin’s count, about 6,500 adults in the U.S. have severe hemophilia A. Of those, the company estimates 2,500 are eligible for Roctavian under the current approval.
"Our findings suggest that gene therapy may reduce the burden of care and improve quality of life in patients with hemophilia B," the authors write.
Relative to site readiness in the U.S., we have identified and are focused on a relatively small number of the largest and most capable hemophilia treatment centers to be ready to treat with ROCTAVIAN at or shortly after launch.