Gene therapy increases factor VIII activity, reduces bleeding in severe hemophilia A

Valoctocogene roxaparvovec induced endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis in patients with severe hemophilia A, according to study results.
Researchers reported findings of the open-label, single-group, multicenter phase 3 study in The New England Journal of Medicine.
“I believe there is an unmet need in men with severe hemophilia A,” Margareth C. Ozelo, MD, PhD, professor of hematology and transfusion medicine in the department of internal medicine at the school of medical science at

Valoctocogene roxaparvovec induced endogenous factor VIII production and significantly reduced bleeding and factor VIII concentrate use relative to factor VIII prophylaxis in patients with severe hemophilia A, according to study results.
Researchers reported findings of the open-label, single-group, multicenter phase 3 study in The New England Journal of Medicine.
“I believe there is an unmet need in men with severe hemophilia A,” Margareth C. Ozelo, MD, PhD, professor of hematology and transfusion medicine in the department of internal medicine at the school of medical science at