People in France with severe hemophilia A who switched to Elocta (efmoroctocog alfa) — an extended half-life replacement therapy — had fewer infusions and a reduced rate of joint bleeds, a real-world study reports. Notably, those who switched to Elocta had a higher bleeding rate, including joint bleeds,…
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A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production of factor VIII (FVIII),  effectively preventing spontaneous bleeds in people with severe hemophilia A, a Phase 1/2 trial shows. Two of the six patients treated to date in this clinical study, both given the…
SerpinPC, a potential treatment for all types of hemophilia, from the drug development pipeline at Centessa Therapeutics, was found to be safe and to reduce patients’ bleeding episodes for up to 1.5 years. In fact, the therapy candidate has the potential to nearly “zero out” the number of…
Nuwiq (simoctocog alfa) is an approved therapy that works to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
Creating personalized hemophilia B care is an important part of hemophilia B management.Your healthcare team can set up a treatment plan based on your specific needs and the severity of your symptoms.
Rare Disease Day, observed this year on Feb. 29, serves as a poignant reminder of the challenges facing those who live with uncommon medical conditions. According to the event’s website, a rare disease is defined as one that affects fewer than 1 in 2,000 individuals. Among these conditions…
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FDA Awards Fast-track Status to Sangamo’s Investigational Gene Therapy SB-525 for Hemophila A
The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic liver-specific promoter to the cell’s nucleus by using recombinant adeno-associated virus (rAAV). The…
Labor and delivery in women with hemophilia Planning for childbirth depends on the needs of the mother and the child affected by hemophilia. Levels of clotting factors should be measured in the last trimester of pregnancy, since it is difficult to measure during labor. If levels are low, treatment may…