Serious bleeds are often a fatal complication in people with hemophilia if not managed early, a 10-year study from Senegal shows. These findings highlight the need for prompt diagnosis of severe bleeding and early administration of replacement therapy to lower the risk of mortality. In resource-limited countries, increasing patients’ and…
News
Two treatment options for hemophilia A are now available to patients in Canada: Esperoct (turoctocog alfa pegol) and Zonovate (turoctocog alfa), marketed by Novo Nordisk. The therapies have been accessible through Canadian Blood Services since April 1. “As part of our continued commitment to provide support to Canadians…
Supporters of World Hemophilia Day, on April 17, are hoping to raise awareness about the bleeding disorder with events planned globally and on social media, and to advocate for sustainable and equitable access to care and treatment. The annual event, which is organized by the World Federation of Hemophilia…
The U.S. Food and Drug Administration (FDA) has given fast track designation to ASC618, an investigational one-time gene therapy for hemophilia A in the pipeline of ASC Therapeutics. Fast track status works to speed the development and regulatory review of treatments for serious disorders that show a potential to fill…
The European Medicines Agency (EMA) has agreed to review a request by CSL Behring to approve the potential gene therapy EtranaDez (etranacogene dezaparvovec) for people with hemophilia B. The request, in the form of a marketing authorization application or MAA, will be reviewed under the EMA’s accelerated assessment, meaning…
Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…
Structural joint damage and significant pain are evident in the ankles of people with hemophilia, but the two were not found to be related to one another in a recent study. Researchers suspect that ankle pain may originate in the somatosensory nervous system, which relays nerve signals from the…
Hemophilia patients with a low number of red blood cells and reduced coagulation factor levels — the activity level of proteins essential for blood clotting — are at a higher risk of increased blood loss during a knee replacement surgery, a study reported. The study, “Risk…
Treatment with the experimental gene therapy Roctavian (valoctocogene roxaparvovec) significantly increased factor VIII levels in men with severe hemophilia A treated in the Phase 3 GENEr8-1 trial and with reasonable safety, findings at one and two years post-treatment show. While declines in these levels were evident over time,…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
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