News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

A modified version of the coagulation protein factor X was able to significantly reduce bleeding in a mouse model of hemophilia A, and may offer a way of treating people with this disease due to factor VIII deficiency, a study suggests. Lab work also showed a potential for treating people…

Ease of use, fear of the unknown, and healthcare providers’ advice are all major factors that play a role in whether people with hemophilia want to switch to new treatment options, a small study suggests. The study, “Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study,”…

Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up…

The U.S. Patent and Trademark Office has granted uniQure a third patent covering the formulation and use of the company’s factor IX Padua gene therapy for the treatment of hemophilia and other bleeding disorders. The new patent (10,465,180) will broaden uniQure’s exclusivity rights over the FIX-Padua encoding gene,…

A natural anticoagulant protein called tissue factor pathway inhibitor (TFPI) is critical for defining the bleeding severity profiles of people with hemophilia A and B, researchers in France report. This discovery, which was found not to hold for healthy individuals, adds support to TFPI as an important element…

A single injection of uniQure‘s gene therapy AMT-061 (etranacogene dezaparvovec) is well-tolerated, effectively increases the levels of clotting factor IX (FIX), and completely prevents bleedings without the need for additional FIX injections in people with hemophilia B, even in the presence of neutralizing antibodies against the treatment,…

An interview-based study from Japan found professional support was necessary when mothers inform their daughters they might be carriers for hemophilia, as the women found themselves “not knowing what to do” and expressing “anxiety due to uncertainty.” In Japan, parents are often responsible for telling a child that a genetic…

Older patients with hemophilia have lower rates of polypharmacy, or use of multiple medications, and of drug-drug interactions than people of the same age without the disease, a review study reports. This is likely due to their regular risk assessments at hemophilia treatment centers (HTCs) and coordination of care…

Direct‐acting antiviral (DAA) therapy is especially effective at eliminating hepatitis C virus (HCV) in people with bleeding disorders, including hemophilia, a study has found. The study, “Elimination of hepatitis C virus infection in patients with haemophilia in Belgium: A single‐centre experience,” was published in Haemophilia.