Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct. The U.S. Food and Drug Administration (FDA) approved N8-GP, or…

SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual bleeds, according to preliminary data from an ongoing Phase 1/2 trial. The trial (NCT03003533) is intended to explore the safety and efficacy of a single intravenous…

A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled in uniQure’s ongoing Phase 2b trial, the company announced. Ultimately, this trial’s purpose is to confirm the dose of AMT-061…

Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number of bleedings and enables more patients to stop having bleeds, according to the first results of a Phase 3b/4 study by Takeda.

My mighty warrior Caeleb is about to start a new chapter in his life with hemophilia; it’s one I never imagined I’d see. About nine months ago, Caeleb began a new treatment with a medication that has been taking the bleeding disorder world by storm. Hemlibra (emicizumab-kxwh)…

A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…

People with hemophilia may benefit from physiotherapy and rehabilitation programs intended to manage bleeding and joint disease (arthropathy), but more research is needed to better understand those programs — and their duration — that might be of most help, a review study says. The review, “Recent advances in musculoskeletal…

Despite having lower oral health scores, children with hemophilia showed no differences in oral health-related quality of life compared to children without the condition, a study found. The study, “Parent’s report on oral health‐related quality of life of children with haemophilia,” was published in Haemophilia. Hemophilia is a…

Thank goodness for the great friendships that we forge along the way. Those of us who live with chronic illnesses learn the most about caring for our loved ones from those who are in our communities. While teaming with medical staff is crucial to the diagnosis and treatment…