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ISTH 2023 | Safety of valoctocogene roxaparvovec for patients with hemophilia A and HIV infection

Margaret Ragni, MD, MPH, University of Pittsburgh, Pittsburgh, PA, discusses the findings of a study investigating the safety of valoctocogene roxaparvovec, an AAV-based gene therapy, for patients with severe hemophilia A and HIV infection. Prof. Ragni highlights that high-grade toxicities were observed in patients who were HIV+ and taking hepatotoxic drugs, and summarizes her recommendations based on these findings. This interview took place at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH), held in Montréal, Canada.

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Consulting and research grants – BioMarin, Sanofi, Spark, Takeda; consulting for BeBio Inc. and for HEMAB, ICER; Serve on Advisory Bd FWGBD; Serve on Editorial Bd for Blood Advances.