News

The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of increasing levels…

The hemophilia team and design experts at Nationwide Children’s Hospital partnered with students from Ohio State University’s Advanced Computing Center for the Arts and Design (ACCAD) to develop a virtual reality game that aims to help the hospital’s pediatric hemophilia patients during procedures. Hemophilia patients often have to go through…

Researchers from the Republic of Korea developed a new type of needle that is able to completely prevent bleeding following syringe needle puncture. This new type of needle can be invaluable for people with blood clotting disorders like hemophilia where syringe injections can have significant side effects such as uncontrolled bleeding.

Considerable progress has been made over the last 50 years in treating and caring for people with hemophilia but more is needed, according a large-scale analysis of men with this disease in the U.S. The report concluded that continued surveillance to monitor and inform hemophilia interventions and patient outcomes is essential,…

Treatments with anti-coagulation drugs such as Brilinta (ticagrelor) may trigger acquired Hemophilia A (AHA) in adults. This finding was reported by Italian doctors after a patient on Brilinta was hospitalized with a hemorrhage. The study, “Acquired Hemophilia A May Be Associated with Ticagrelor Therapy in a 52-Year-Old Man After a…

Japan’s Ministry of Health, Labor and Welfare has approved CSL Behring‘s Idelvion (albutrepenonacog alfa) for the prevention of bleeding tendency in patients with hemophilia B. Hemophilia B, a bleeding condition that mainly affects males, is characterized by a deficient or defective factor IX protein, and is treated with intravenous infusion of factor IX.

Scientists have improved protein drugs using an approach based on “ancestral sequence reconstruction,” or ASR. The newly established strategy promises to enhance the ability to engineer more efficient and durable proteins, as in the case of human factor VIII for hemophilia. The study “Enhancing the pharmaceutical properties of protein drugs by…

Swedish Orphan Biovitrum AB recently announced that Eloctate (efmoroctocog alfa; brand name Elocta in Europe), a treatment for hemophilia A, has been approved for reimbursement in the United Kingdom, Italy, and France. These countries join others in the European Union — Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands,…

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.

Researchers reviewed the use of high-purity concentrates containing two plasma molecules that are lacking in patients with Von Willebrand disease (VWD), a hemorrhagic disorder. These products may be a good strategy for managing pediatric patients with VWD and hemophilia A (HA) who do not respond to other treatments. The study, “Human Von…