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,…

Being unemployed or under-educated can have a negative effect on hemophilia patients’ quality of life, including their pain levels and ability to function, according to the results of a questionnaire that were presented at the ASH Annual Meeting and Exposition. In the patients surveyed, increased pain, functional impairment and reduced…

The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion date…

Catalyst Biosciences will present three posters with data from its two investigational candidates for hemophilia at the upcoming 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) Feb. 1-3 in Paris, France. The first poster presentation is for a next-generation factor VIIa, marzeptacog alfa (activated), that showed…

People with hemophilia may benefit from mild exercise to minimize their risk of bleeding according to a study published in the scientific journal Haemophilia.