Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…

Two new mutations affecting the production of functional clotting factor VIII were identified in a group of Vietnamese patients with hemophilia A, researchers report. Their findings were described in a letter to the editor, “Mutation characteristic of 103 haemophilia A patients in Vietnam: Identification of novel mutations,”…

The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…

Hemophilia care providers in the U.K. have insufficient knowledge on how to assess the risk of prolonged bleeding associated with dental procedures, a study has found. The study, “U.K. hemophilia treaters’ knowledge of risk assessment for prolonged bleeding associated with dental procedures,” was published in the journal…

A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…

Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…

Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and adult males with severe hemophilia A and without inhibitors, according to a real-world study. The study, “Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective…

People with severe hemophilia A living in Europe can now receive treatment with Hemlibra (emicizumab-kxwh) to prevent bleeding episodes. The European Commission approved the prophylactic use of Hemlibra in patients of all ages who do not have factor VIII inhibitors, announced the treatment’s manufacturer, Roche. Hemlibra is a man-made antibody…

More than half of adults who either have hemophilia or are family members affected by the disease support the use of preconception and prenatal genetic screens, a survey reveals. However, 90 percent of this population do not agree with ending pregnancies due to hemophilia, the results also showed. Findings…

Erectile dysfunction, more commonly known as male impotence, is associated with older age in men with hemophilia, especially those who smoked long term and have high blood pressure, a study reports. The study, titled “Erectile dysfunction in patients with haemophilia,” was published in Haemophilia. Hemophilia is…