Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. Generation Bio has announced new data showing that its investigational non-viral gene therapy platform can increase levels of the clotting…
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Bayer’s Jivi, an FVIII replacement therapy for hemophilia A, has a longer half-life and a slower clearance from blood circulation than Eloctate, and may protect against bleeding for longer periods, results of a Phase 1 trial in people with severe disease suggests. The study “Direct comparison of…
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Living With Hemophilia: Brandston’s Story
To help prevent bleeds in hemophilia patients, many people living with the disease need to have infusions of clotting factor. In this video from infuzrTV, we meet super cute 5-year-old Brandston. Brandston has severe hemophilia A and needs to have clotting factor infusions on alternate days. MORE: …
Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…
A team led by Lurie Children’s Hospital of Chicago researchers reported a case study of a newborn with combined spontaneous splenic rupture and severe hemophilia A. This baby, whose case is believed to be the first documented, was successfully treated without surgical intervention. Results from the study, “Successful medical management of a…
A number of clinical studies have reported that bone marrow-derived cells contribute to the production of clotting factor VIII (FVIII), the molecule linked to hemophilia A. Because the current treatment for this bleeding disorder is associated with some disadvantages and risk, hematopoietic stem cell transplant (HSCT) has become an emerging therapy approach…
Altuviiio (efanesoctocog alfa) is an approved long-lasting factor VIII (FVIII) replacement therapy used to reduce the frequency of bleeding episodes or control and manage active bleeds, including those occurring during surgery, in children and adults with hemophilia A.
Hympavzi (marstacimab) reduced bleeding rates and improved quality of life in adults and adolescents with hemophilia A or B who have inhibitors (antibodies against standard replacement therapies). That’s according to final data from the Phase 3 BASIS study (NCT03938792), which demonstrated the treatment met its main goal of…
As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia A or BÂ regardless of inhibitor status, early results from an extension study show. The findings were presented at the recent World Federation of Hemophilia Virtual Summit and…
Ruptured spleens are rare in newborns, usually occurring secondary to an underlying coagulation disorder, such as hemophilia, or a splenic abnormality. Here, researchers report the first case of a newborn with a spontaneous splenic rupture and severe hemophilia A who was successfully treated without surgical intervention. The case study, “…