Some hemophilia patients may, over time, develop inhibitors which restrict the efficacy of treatment making bleeding episodes more difficult to stop. MORE: Intracranial hemorrhage risk – when to see a doctor According to the Centers for Disease Control and Prevention (CDC), although the majority of people living with…
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A gene therapy that can treat patients with severe hemophilia A is likely to be more cost-effective over the long term than reliance on prophylactic (preventive) therapy using factor VII (FVIII), a model-based analysis of the two treatment approaches reports. The study, “Gene therapy in hemophilia A: a cost-effectiveness…
Scientists have developed advanced liver organoids — lab-grown “mini-organs” that mimic the function of organs in the body — that are capable of growing their own functional blood vessels and producing clotting factors that could eventually be used to treat hemophilia, according to a study. In a mouse model…
Biogen and Sobi announced that they will be presenting updated and long-term data on two approved hemophilia treatments, Eloctate (marketed as Elocta in Europe) for hemophilia A, and Alprolix for hemophilia B, at the World Federation of Hemophilia (WFH)’s 2016 World Congress next week. Specifically, data from separate Phase 3 extension studies evaluating each treatment’s safety and…
The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…
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How to Explain Hemophilia to Children
In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…
A real-world study in Spain found that, among hemophilia patients, those with hemophilia A may have have worse joint health — and more joint damage, particularly in the ankles — than those with hemophilia B. Importantly, though, such damage appeared to be less frequent for individuals on regular…
Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…
“There are only two ways to live your life. One is as though nothing is a miracle. The other is as though everything is a miracle.” — often attributed to Albert Einstein I live with a bleeding disorder, hemophilia A. Survival often is truly about attitude, optimism, and perseverance.
Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will…