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hemophilia A

30th Jul 2025

Plain language summary: 4-year outcomes from testing valoctocogene roxaparvovec gene therapy in people with hemophilia A

21st Mar 2025

A plain language summary of the two-year results from a five-year study on giroctocogene fitelparvovec gene therapy for individuals with severe hemophilia A

17th Mar 2025

A plain language summary comparing bleeding outcomes after valoctocogene roxaparvovec gene therapy versus emicizumab prophylaxis

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.
14th Nov 2023

Plain Language Summary of Publication article: the GENEr8-1 study of valoctocogene roxaparvovec gene therapy for hemophilia A

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