In healthy people, a protein called factor VIII (FVIII) helps blood to clot and prevents excessive bleeding. People with hemophilia A lack FVIII because a faulty F8 gene is giving the wrong instructions to the liver cells that make it. Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy designed to transfer working copies of the F8 gene into liver cells. This summary describes the GENEr8-1 study, which looked at how well valoctocogene roxaparvovec works for treating people with severe hemophilia A compared with their usual FVIII replacement therapy, and its safety. 134 men received valoctocogene roxaparvovec; results from the first 2 years are reported.This Plain Language Summary of Publication article from Future Rare Diseases focusses on the a blood clotting disorder called Haemophilia A. The summary describes how a therapy called Valoctocogene roxaparvovec (ROCTAVIAN™) significantly improved bleed control in men with severe hemophilia A.

Visit the Future Medicine site using the link to read the article.

The original article on which this summary is based is called ‘Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A’ was published in the New England Journal of Medicine. 

Visit the New England Journal of Medicine site using the link to read the article.