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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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…
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.
When people hear the word “hemophilia,” they usually picture bleeds, factor infusions and other treatment routines, and trips to the hospital. Those things certainly shape the condition, but they’re not the whole story. Over the years, I’ve noticed something that feels harder to explain, yet…
Ixinity (recombinant factor IX product, IB1001) was found to be safe and effective in previously treated children with hemophilia B younger than 12, according to data from Aptevo Therapeutics‘ ongoing Phase 3 clinical trial. The company presented data from the analysis of two studies at the recent Hemostasis and Thrombosis…
US Bioservices, a specialty pharmacy, announced that it is working with the pharmacy benefit management company MedImpact to dispense prescriptions and select services through the MedImpact Direct Specialty Program for the hemophilia community. Under the collaboration, US Bioservices will offer hemophilia patients who are MedImpact members the opportunity to…
Despite having lower oral health scores, children with hemophilia showed no differences in oral health-related quality of life compared to children without the condition, a study found. The study, “Parent’s report on oral health‐related quality of life of children with haemophilia,” was published in Haemophilia. Hemophilia is a…
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Promising Interim Results Reported for uniQure’s Gene Therapy AMT-061 in Phase 2b Hemophilia B Trial
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,…