WFH 2020: Four-year data show that type A haemophilia gene therapy valoctocogene roxaparvovec can continue to reduce bleeding
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Last Update: 2020-07-12
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Source: Internet
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Author: User
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BioMarin, a pharmaceutical company, recently released the results of an ongoing Phase I/II trial, which for four years showed a significant reduction in bleeding rates and improved levels of factor VIII in patients with severe haemophilia type A patients following the infusion of experimental gene therapy valoctocogene roxaparvovecLead researcher John Pasi of BioMarin presented the findings at the World Hemophilia Consortium (WFH) summitThe results presented on WFH included four-year data for the 6e13 vg/kg queue and three-year data for the 4e13 vg/kg queueBioMarin noted that all of the study participants had severe haemophilia type A at baseline6e13 vg / kg in 6 patients who had previously received the Factor VIIIPrevention, and in the four years following treatment with valoctocogene roxaparvovec, the cumulative average annual bleeding rate (ABR) decreased by 95% to 0.8 compared to the pre-infusion baselineIn the fourth year, the average ABR was 1.3, compared with 16.3 in the year before treatmentBioMarin noted that 86% (6/7) of all 7 study participants in the 6e13 vg/kg queue had no bleeding after four years
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