Bleeding rates and the use of replacement therapies among men with severe or moderately severe hemophilia B remain low at least three years after a single dose of the approved gene therapy Hemgenix (etranacogene dezaparvovec). That’s according to updated data from the Phase 3 HOPE-B clinical trial…
Adding an antibody targeting the anticoagulant protein S to factor IX (FIX) replacement therapy accelerated blood clotting in samples from children with hemophilia B, including those with severe disease, a study showed. Greater production of the blood clotting protein thrombin also was seen in these samples, while blocking…
Changes in the cellular microenvironment could influence immune responses against factor VIII (FVIII) replacement therapy in people with hemophilia A, a new study suggests. Data from the cell culture study showed that FVIII proteins that cause an immune reaction differ depending on which other molecules are present in…
Prophylactic, or preventive, treatment with an experimental replacement therapy called TQG202 safely maintained a low bleeding rate in people with severe hemophilia A. That’s according to new data from a Phase 3 clinical trial reported in a recent study, which showed TQG202 worked to control bleeds in testing against…
Serious bleeds are often a fatal complication in people with hemophilia if not managed early, a 10-year study from Senegal shows. These findings highlight the need for prompt diagnosis of severe bleeding and early administration of replacement therapy to lower the risk of mortality. In resource-limited countries, increasing patients’ and…
Replacement therapy is one of the standard therapies used to treat hemophilia, a genetic disorder caused by the absence or defects in blood clotting factors. Hemophilia makes patients highly susceptible to bleeding due to a compromised cascade of clotting reactions that are necessary to “seal” injuries.
Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…
Hemophilia A patients age 12 and older who live in Canada now may receive prophylactic (preventive) treatment with Bayer’s antihemophilic factor Jivi. Health Canada approved Jivi as a treatment to prevent or reduce the frequency of bleeding episodes in patients age 12 or older who have received…
Preliminary results of a Phase 1/2a trial of the hemophilia A experimental treatment BIVV001 show a significant extension of replacement therapy lifetime in the blood, Bioverativ announced. The ongoing, and still recruiting, open-label, multicenter EXTEN-A study (NCT03205163) is evaluating the safety, tolerability, and pharmacokinetics of a single…
Scientists managed to treat hemophilia B using a new method of protein replacement therapy in a mouse model of the condition.
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