In our quest for security and certainty, it’s only natural to gravitate toward options that offer zero risk. Think about it: When you purchase insurance or opt for a product with a money-back guarantee, you’re essentially seeking assurance against any potential loss. This tendency to avoid risk at all…
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Nuwiq, a fourth-generation genetically engineered version of blood-clotting factor VIII, is a safe and effective way of controlling bleeding in people with severe hemophilia A during and after surgery, a study reports. The research, “Efficacy and safety of Nuwiq® (human-cl rhFVIII) in patients with severe haemophilia A undergoing…
Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.
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Pfizer’s Expansion in North Carolina May Help Advance Manufacturing of Hemophilia Gene Therapies
Pfizer will invest an additional $500 million into the construction of its new manufacturing facility in Sanford, North Carolina, the company announced. According to the company, the facility could help advance the manufacturing of one-time gene therapies that use modified adeno-associated viral vectors, such as SB-525, which is…
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Gene Therapy Seen to Produce FVIIa Levels Needed to Control Bleeding in Rat Study of Hemophilia A
A gene therapy-based factor VIIa (FVIIa) given as prophylactic, or preventative, treatment was seen to produce specific factor levels needed to reduce the frequency of spontaneous bleeding episodes in a rat model of hemophilia A. According to scientists, this work may be a first step in developing therapies that…
Treatment with Eloctate and Alprolix improves joint health, bleeding episodes and quality of life of patients with hemophilia, Bioverativ and Sweden’s Sobi recently announced. The data is being presented at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress,  through July 13 in Berlin, Germany. Hemophilia is a…
Administering low doses of Feiba, or activated prothrombin complex concentrate (aPCC), as a prophylactic (preventive) treatment after acute bleeding events reduces the risk of relapse in patients with acquired hemophilia A, a study shows. The study “Low dose of IPCC after the initial treatment in acquired…
When I was a kid in the 1970s and ’80s, I thought children who had trampolines in their yards were amazing. Being invited over to jump on a friend’s trampoline was a special treat. Later, as a teacher, I lost track of how many of my students acquired injuries from…
Shellye Horowitz shares her story of working through the ups and downs of hemophilia, from carrying factor to school every day to creating safety plans and finding supportive colleagues. She reflects on dreaming big, adapting with confidence, and the belief that hemophilia doesn’t…
Aptevo Therapeutics provided an update on Ixinity, its hemophilia B therapy, that includes developing a larger 3,000 IU vial assay for patient convenience. The company also plans to initiate a Phase 4 clinical trial in a pediatric patients, and to apply for Ixinity’s approval outside of the United States.