Save One Life, the first globally focused organization to sponsor children with bleeding disorders one-on-one, recently celebrated its 15th anniversary. More than 130 guests from the hemophilia community worldwide gathered in New York City Sept. 29 at the Manhattan Penthouse on Fifth Avenue. The guest of honor was…

A publicly available, patient-reported outcome measure is being jointly developed by Green Park Collaborative (GPC) and the National Hemophilia Foundation (NHF) to evaluate the effect of gene therapy on the mental health outlook of people with hemophilia type A and type B. The collaboration follows work…

Switching to Hemlibra (emicizumab) can add more than $100,000 a year to healthcare costs for people with hemophilia A in the U.S. who were previously using preventive replacement therapies, according to a new analysis. “Increased use of [Hemlibra] may cause an increase in economic burden for payers without…

Hemophilia A patients with factor VIII (FVIII) inhibitors show deficiencies in regulatory B- and T-cells — immune cells that typically dampen immune and inflammatory responses — but their levels can be restored with successful immune tolerance induction (ITI), a study shows. These findings shed light on how ITI — given…

Researchers investigated self-reported health-related quality of life (HrQoL) in children and adolescents with hereditary bleeding disorders, such as hemophilia A, and found no differences between patients and their healthy siblings and peers. The research paper, titled “Health-Related Quality of Life in Children and Adolescents with Hereditary Bleeding Disorders…

High levels of antibodies against clotting factor VIII (FVIII) were associated with a slower response to immunosuppressive treatments in a small group of people with acquired hemophilia A in new study in China. Of 34 patients, 29 achieved a complete response to their first-line immunosuppressive therapy. Still, a lower…

Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more expression, or activity, of genes involved in activating the immune system, a small study reports. “The results of our study reveal that there is an upregulation of genes involved with…

A few weeks ago, my oldest son, Julian, called me from the Blue Ridge Mountains in North Carolina and informed me that he had throbbing pain in one of his teeth. He immediately went to a nearby dentist and got news he didn’t want to hear: The tooth…

Novo Nordisk has resumed its Phase 3 clinical trials investigating concizumab as a potential treatment for hemophilia A and B, the company announced in a press release. The trials were paused in March due to the occurrence of non-fatal thrombotic (blood-clotting) events in three patients. In collaboration…