The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B —  in Priority Medicines (PRIME), a voluntary European regulatory initiative to support medicines that target an unmet medical need. PRIME aims to optimize development plans and speed up evaluation so that these priority medicines can…

My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in 2018.

Adult hemophilia patients use hospital emergency departments mainly due to cardiovascular events, while pediatric patients most frequently complain of injuries, according to researchers. U.S. emergency department (ED) visits by hemophilia patients had a national estimated cost of $60 million in 2012, the year studied. These observations resulted from the retrospective and…

A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that characterizes the disease in a mouse study. This potential new method might be a viable alternative for many clotting disorders. The study, “Systemic delivery of Factor IX messenger RNA…

Psychological distress triggers microscopic changes in regions of hemophiliac children’s brains that deal with emotions and cognition — and the changes show up before abnormal results on neuropsychiatric tests, researchers discovered. The team said larger studies are needed to confirm the links they found between brain changes, the stage of the…

Scientists managed to treat hemophilia B using a new method of protein replacement therapy in a mouse model of the condition.

Researchers have developed a protein drug that may teach the body to tolerate, rather than reject, the clotting factor treatment given to hemophilia patients. The new drug, tested in dogs, may lead to novel human therapies. The study, “Oral Tolerance Induction in Hemophilia B Dogs Fed with Transplastomic Lettuce,” appeared in the…

Therapy to prevent bleeding (called prophylaxis) in patients with severe hemophilia A led to decreased joint bleeding when it was started at any age. However, improved joint motion was only effective if prophylaxis was started before age 4 in non-obese patients, according to researchers. The study, “Prophylaxis Usage, Bleeding…

Men with hemophilia who have a Medicaid health plan spend less on healthcare than those with employer-sponsored health insurance, due to lower reimbursement. However, Medicaid spends more on extensive healthcare users than employer-sponsored plans, according to a new study from the Centers for Disease Control and Prevention (CDC). The study,…

A new, modified version of recombinant factor VIII (rFVIII), called rVIII-SingleChain, showed great efficacy and safety in treating bleeding events in children with severe hemophilia A. The study, “Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial,” was published in…