Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3 clinical trial were completely free of bleeds over about one year of treatment. That’s according to new findings from the recently-completed XTEND-Kids study (NCT04759131), which were presented as late-breaking…

Nearly two-thirds of children with severe hemophilia A on preventive, or prophylactic, treatment with once-weekly Altuviiio (efanesoctocog alfa) in the yearlong XTEND-Kids study were free of bleeding episodes needing treatment. More than 80% in this Phase 3 clinical trial also were free of spontaneous bleedings and joint bleeds needing…

Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s…

The effectiveness of liver-directed, viral-based gene transfer — the most used experimental gene therapy approach for hemophilia A — is significantly reduced in mouse models of hemophilia A relative to healthy mice, a study shows. This limitation was associated with a pronounced reduction in fenestrations — cell surface…

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…

Prophylactic replacement therapy to prevent bleeds was more cost-effective than on-demand treatment of active bleeds for children and adolescents in China with moderate or severe hemophilia A without inhibitors, a study reported. The superior cost-effectiveness of prophylaxis over on-demand treatment was mainly driven by the on-demand costs of treating…

Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology. The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete…

When my first son, Julian, was born in 1996 and diagnosed with severe hemophilia A, I thought my world was ending. I expected to give birth to a healthy baby boy. I never dreamed he’d have a medical condition. I went through the stages of grief: denial,…

Children and young adults with hemophilia A and hemophilia B have several behavioral problems, ranging from depression and anxiety to aggressive behaviors, a small study suggests. Age, disease severity, and joint disease duration were found to be significantly correlated with several of these problems. The findings were reported in the…

Boys with severe hemophilia A who switched to Kovaltry (octocog alfa), which replaces a missing clotting protein called factor VIII (FVIII), retained the therapy for longer in the body and had fewer bleeds, a study in China reported. Findings come from a head-to-head comparison of Kovaltry and three…