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First Hemophilia A Patient Enrolls in Phase 4 Trial for Eloctate Immune Tolerance Induction

Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will…

How Kids Can Explain Hemophilia to Their Friends

This children’s video from Centers for Disease Control and Prevention focuses on how to tell your friends that you have hemophilia. MORE: How kids can play safe with hemophilia Billy, a young boy with hemophilia, answers some of the questions that his friends ask about the disease. He…

Eloctate Improves Joint Health in Hemophilia A, Phase 3 Study Shows

Interim results from the Phase 3 ASPIRE study indicate that patients with hemophilia A showed continuous improvement in joint health for nearly three years while receiving prophylactic dosing of Bioverativ and Sobi’s Eloctate. The interim results were reported in a study titled, “Improved joint health in subjects with severe haemophilia A…

How Diet Can Help With Hemophilia Management

Eating well is important for everyone, but particularly for those living with hemophilia. Eating a diet that encourages strong bones and muscles and helps keep you at a healthy body weight is essential. MORE: Young boys explain what it’s like to live with hemophilia Obesity is becoming a major problem when it…

BioMarin’s Investigative Gene Therapy for Hemophilia A Wins FDA Breakthrough Designation

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to treat patients with hemophilia A. This new status is likely to speed up the development and regulatory review of valoctocogene roxaparvovec, which has already received an Orphan Drug Designation from both…

Could Genome Editing Be a Future Treatment for Hemophilia?

An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA-editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA, removing…