Hemgenix (etranacogene dezaparvovec) for hemophilia
Hemgenix (etranacogene dezaparvovec), formerly known as AMT-061 and EtranaDez, is a gene therapy given as a one-time intravenous (into-the-vein) infusion to treat bleeding episodes in hemophilia B.
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October 30, 2025
by Margarida Maia, PhD
Giroctocogene fitelparvovec for hemophilia
Giroctocogene fitelparvovec, formerly known as SB-525 or PF-07055480, is a gene therapy being developed to reduce the risk of bleeding episodes in people with hemophilia A.
Joint damage common in Chilean children with severe hemophilia
Half of the children in Chile with severe hemophilia A and B exhibit joint damage despite receiving preventive treatment with clotting factors, a study reports. Damage to the cartilage is the main sign of joint impairment in children older than 8 years, while synovitis, or inflammation of the synovial…
Words matter when we talk about conditions like hemophilia
I’ve lost count of how many times I’ve seen phrases like “victims of hemophilia” or “afflicted with hemophilia” in local news articles. Sometimes the phrase is “suffering from hemophilia.” To many readers, these words might sound sympathetic — even caring. But to those of us…
Eloctate, Alprolix control bleeds in hemophilia patients’ major surgeries
Treatment with the extended half-life (EHL) therapies Eloctate (efmoroctocog alfa) and Alprolix (eftrenonacog alfa) was found to safely and effectively control bleeding during major orthopedic surgeries in people with hemophilia A or B, according to real-world data from two Italian centers. The therapies helped patients maintain…
October 21, 2025
by Bionews Staff
Challenging misconceptions about women and hemophilia
Jennifer Lynne shares her journey with von Willebrand disease and hemophilia B, reflecting on the misconceptions women face, the power of patient documentation, and the importance of community support. She highlights how advocacy has shaped her life and why sharing stories can drive change in Hemophilia and Me.
October 15, 2025
by Marisa Wexler, MS
Profilnine (factor IX complex) for hemophilia
Profilnine (factor IX complex) is a plasma-derived factor replacement therapy approved to prevent and control bleeding episodes in people with hemophilia B.
South American men with severe hemophilia face ‘large burden’
Men in South America with severe hemophilia A or B are hit with both direct and indirect costs related to the genetic disease, which also affect quality of life, a new study reports. As much as 99% of the direct costs of hemophilia were related to factor…
Living in abundance when hemophilia medication is scarce
Scarcity mindset is the belief that something you rely on will one day run out. It’s most often talked about in the context of money, which resonates with me deeply. As a neurodivergent person married to someone with hemophilia, money has always felt like one of my shields…