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…
A foam roller to release tension in the fascia — a casing of fibrous tissue that surrounds and holds other tissues in place — is safe to be used by patients with knee joint disease caused by hemophilia, a Spanish has study found. When the fascia tightens around muscles,…
A multi-institute research collaboration has received a $12 million grant from the National Heart Lung and Blood Institute to understand what’s behind limitations in gene therapies currently being tested in people with hemophilia A. The goal is to use this information to develop safer and long-lasting gene therapies…
Weekly use of the experimental, long-acting therapy efanesoctocog alfa safely and effectively prevents bleeds in adolescents and adults with severe hemophilia A, according to top-line, one-year data from the Phase 3 XTEND-1 trial. The therapy — being jointly developed by Sanofi and Sobi — was also superior to approved preventive therapies…
The first participant has been dosed in a Phase 1/2 dose-confirmation trial that is testing FLT180a, an experimental gene therapy that Freeline is developing to treat hemophilia B. “We are very excited about this progress in our hemophilia B program as our enhanced focus on execution is starting to…
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