Researchers at the Center for Research in Transplantation and Immunology in France have reviewed a paper concerning certain strategies utilized to correct hemophilia B and the importance of gene delivery vehicles in successful therapies for treatment of the condition. The review paper, “Successful correction of hemophilia by CRISPR/Cas9 genome…

Gene therapy delivered by microbubbles shows promise in treating hemophilia B, a hereditary bleeding disorder caused by the lack of blood clotting factor IX, new research suggests. The study, “Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice,” appeared in the journal Gene Therapy. Hemophilia B affects approximately…

Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX (FIX), the clotting protein that is missing or faulty in hemophilia B, leading to about three times fewer bleeding episodes compared with prophylaxis, or preventive treatment, with regular infusions…

The initial treatment in patients newly diagnosed with hemophilia B should definitely be done in hospital according to a study conducted by scientists in Turkey.

Hemophilia has a substantial impact on sexual function and well-being for many men with the disease, but they rarely discuss it with their doctors, a study from the Netherlands found. “To address this gap, tools designed to provide and assess information about sexual functioning and well-being to normalize the…

Direct‐acting antiviral (DAA) therapy is especially effective at eliminating hepatitis C virus (HCV) in people with bleeding disorders, including hemophilia, a study has found. The study, “Elimination of hepatitis C virus infection in patients with haemophilia in Belgium: A single‐centre experience,” was published in Haemophilia.

Time flies so surprisingly fast. It just dawned on me that I’ll be a father in three months; my wife, Cza, is already 26 weeks into her pregnancy. There’s a lot of pressure, but I hope that my kid can be proud to have me as a father. It’s of…

Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…

During my husband Jared’s recent hospitalization, I heard one particular sentence more times than I can count: “We’ll wait for Doc’s approval.” Jared was admitted for a burn on his hand, an injury that, on its own, often follows a fairly standard treatment path. But because he lives with…

Treatment with Alprolix improves bleeding control, reduces the need for infusions, and improves patient compliance among people with hemophilia B, according to a review of real-world data. The study, “Real‐world data demonstrate improved bleed control and extended dosing intervals for patients with haemophilia B after switching…