This Plain Language Summary of Publication article (PLSP) from Future Rare Diseases examined Hemophilia A, a bleeding disorder caused by a non-functional F8 gene, which prevents the body from producing Factor VIII (FVIII), an essential blood-clotting protein. While traditional treatments involve regular FVIII concentrate or emicizumab to prevent bleeding, ROCTAVIAN (valoctocogene roxaparvovec) offers a new approach as the first approved gene therapy for hemophilia A. It works by delivering functional copies of the F8 gene to the liver, enabling patients to produce their own FVIII and reduce excessive bleeding.

Visit the site using this link to read the article.

This PLSP is based on an article called ‘Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial’ and was published in Research and Practice in Thrombosis and Haemostasis.

Visit Elsevier using the link to read the original article.