Last Thursday, I visited Washington, D.C., to join my fellow blood brothers and sisters in lobbying legislators regarding issues that directly affect the bleeding disorders community. I must admit that I was a little nervous. Several times, I wondered, “What in the world am I doing here? I’m only…
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The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s Investigational New Drug (IND) application for its gene therapy candidate to treat severe hemophilia A. FDA’s decision on BMN 270, also known for its generic name valoctocogene roxaparvovec,…
Service dogs are typically thought of as necessary companions for the visually impaired, but service and therapy dogs can be a practical solution for people with a variety of chronic illnesses. As well as being a trusted friend, service dogs can expand owners’ motor abilities, granting them new independence and allowing them…
Should regulatory authorities agree with favorable results from Genentech’s ongoing Phase 3 studies of Hemlibra (emicizumab-kxwh), hemophilia A patients may have a treatment they can use regardless of inhibitor status, with a choice of three dosing schedules that might better match their lifestyle and preferences. “The ultimate goal for…
San Francisco-based Catalyst Biosciences has completed toxicology studies of its new drug CB 2679d/ISU304 — a highly potent next-generation coagulation factor to treat hemophilia B. This follows the therapy’s recent Investigational New Drug (IND) approval, a necessary step before starting clinical studies, by the Korean Ministry of Food and Drug Safety. Patients…
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…
Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…
Becoming a parent changes everything in life. This was certainly true for my husband, Jared, who’d always dreamed of being a father, despite having severe hemophilia B and frequent seizures. When our daughter, Cittie, was born, that dream came to life, giving him more purpose and even improving…
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N8-GP Improves Quality of Life and Satisfaction of Children with Severe Hemophilia A, Study Finds
Researchers have found that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.
Problems at work, limited engagement in recreational activities, and less relationship satisfaction contribute to a lower health-related quality of life (HRQoL) and greater pain severity, as reported by adults with hemophilia B, according to a study. Findings also showed that anxiety and depression in caregivers of children with this…