Tag: Roctavian

In clinical trials, a single infusion of valoctocogene roxaparvovec reduced the mean annualized bleeding rate by 83.8% compared to prior Factor VIII prophylaxis [1.10.3]. This one-time gene therapy is transforming treatment, but how is valoctocogene roxaparvovec administered to achieve such significant results?

According to clinical trial data, valoctocogene roxaparvovec (brand name Roctavian) demonstrated a significant reduction in annualized bleeding rates for men with severe hemophilia A. This innovative treatment represents a major advancement in the management of this genetic bleeding disorder by addressing its root cause through gene therapy.