The benefits of a one-time gene therapy were sustained for up to 13 years among men with severe hemophilia B with no new safety concerns emerging, according to newly published results from a clinical trial. The findings, which encompass the longest ever follow-up for a gene therapy study in…

Scientists managed to treat hemophilia B using a new method of protein replacement therapy in a mouse model of the condition.

Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled from 2005 to 2019, an analysis reported. Its researchers expect this finding will help state agencies with decisions regarding treatment coverage based on their Medicaid budget, given that the program…

Regulatory agencies in the U.S. and Europe are reviewing applications seeking the approval of fidanacogene elaparvovec, an experimental one-time gene therapy being developed by Pfizer for the treatment of hemophilia B in adults. The U.S. Food and Drug Administration (FDA) expects to complete the review process and…

Beqvez (fidanacogene elaparvovec-dzkt), a discontinued gene therapy for hemophilia B, was shown to help reduce joint bleeding in Phase 3 clinical testing. That’s according to new analysis results from an ongoing global study that assessed the treatment’s effectiveness and found positive results. Specifically, “through 15 months, 12 of…

Over the past few years, I have had the pleasure of connecting with many women who are diagnosed with hemophilia A, hemophilia B, or as symptomatic carriers. These women have so many stories to share. The thread of commonality in their journeys is their struggle…

Adults with moderate-to-severe hemophilia engage in less physical activity and have reduced physical fitness relative to healthy adults, according to a recent study. Data also indicated these patients had more indicators of abdominal fat accumulation and more often had high blood pressure than healthy people, which could overall reflect…

Men in South America with severe hemophilia A or B are hit with both direct and indirect costs related to the genetic disease, which also affect quality of life, a new study reports. As much as 99% of the direct costs of hemophilia were related to factor…

In hemophilia A, the most significant therapeutic complication is the development of antibodies against factor VIII (FVIII) that inhibit its coagulant activity and make the therapy ineffective. The plasma-derived porcine FVIII product represents a therapy with low levels of autoantibodies and successful management of bleeding when compared to human plasma-derived products.