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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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.
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…
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…
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…
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.
Last in a series. Read parts one and two. For 21-year-old Domenic Catrine, hemophilia B once dictated every part of life, from wearing a helmet during childhood playtime to college routines shaped by weekly factor infusions. Then he underwent gene therapy during his sophomore year in…
Hemophilia has a substantial impact on sexual function and well-being for many men with the disease, but they rarely discuss it with their doctors, a study from the Netherlands found. “To address this gap, tools designed to provide and assess information about sexual functioning and well-being to normalize the…
Profilnine (factor IX complex) is a plasma-derived factor replacement therapy approved to prevent and control bleeding episodes in people with hemophilia B.