I am a woman with mild hemophilia. I was angry when I realized that I had gone years without proper diagnosis and treatment. Over the years, I have had multiple bleeds that would have healed more effectively with access to factor replacement products. Many people mourn…

Proper nutrition is lacking in almost half of children with hemophilia in Iraq, with a significant portion of these children underweight compared to peers and a sizable number overweight, a study of pediatric patients in an Iraqi province found. The work also showed an association between low levels of parental education…

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…

I am writing to you on behalf of all the women with a hemophilia mutation. My blood sisters have varying medical diagnoses: asymptomatic carrier, symptomatic carrier, mild hemophilia, moderate hemophilia, and even severe hemophilia. Their diagnosis is often linked to the philosophies of their hematologist or hemophilia treatment center.