Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie…
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The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…
A study presented at American Society of Hematology’s (ASH) annual meeting and exposition shows that Adynovate is effective for the prevention and treatment of severe pediatric Hemophilia A (HA). Eric Mullins, MD and his colleagues from the Cancer and Blood Diseases Institute at Cincinnati Children’s Hospital Medical Center presented their work…
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…
Hemophilia is sometimes a surprise in families. Research shows that approximately 30% of cases of hemophilia A and B are sporadic, meaning there’s no known family history. “This happens when a new alteration in the F8 or F9 gene occurs by chance in an egg cell or…
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Data Supports Potential for Personalized Hemophilia A Therapy with Nuwiq, WFH Presentations Show
Since every hemophilia A patient responds differently to therapies, researchers are working to develop personalized preventive (prophylaxis) strategies with Nuwiq (antihemophilic factor [recombinant].) Octapharma, the treatment’s manufacturer, presented data showing that an individual’s pharmacokinetic (PK) profile (how the therapy works once inside the body) can be used to possibly…
Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…
AMT-060 was a first-generation gene therapy for hemophilia B that was tested in clinical trials as a potential treatment for reducing the risk of bleeding episodes in patients.
Researchers have identified four distinct subgroups of hemophilia A patients according to their profile of factor VIII-targeting antibodies. This may help predict those at risk for developing inhibitors that halt the efficacy of FVIII replacement therapy. The results were presented in a poster titled “Data Coming out of the…
Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the DalcA Phase…