Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes. Hemgenix is now the first and only gene therapy available to this patient group. “The approval of Hemgenix in Canada…
This year’s Hope Conference, a national educational event presented by Hope Charities for the bleeding disorders community, is set for Nov. 2-4 in Florida, with an online option as well. The annual conference, to be presented at the Baytowne Conference Center, in Miramar Beach, is designed to bring together…
People with hemophilia are living longer, but Black males still are more likely to die from the disease, and at a younger age, compared with white people, a U.S. study finds. More research is needed to understand the reason for this difference and to improve care and survival rates…
Venture philanthropy fund Pathway to Cures (P2C)) has invested $500,000 in Five Liters, which is developing noninvasive, nonpharmacological neuromodulation therapies for treating inheritable blood and bleeding disorders, such as hemophilia and von Willebrand disease (VWD). “Investing in Five Liters’ early-stage research is a critical step in addressing unmet…
Two longtime bleeding disorders advocates, who are the parents of a son with hemophilia B, have made a founding donation to Pathway to Cures — dubbed P2C — the new venture philanthropy fund of the National Bleeding Disorders Foundation (NBDF). The transformational gift was added to the estate…
A global survey of more than 2,700 hemophilia patients, caregivers, and healthcare providers, which aimed to uncover the daily challenges of living with the rare blood disorder or caring for those who do, found that many patients regularly hide their symptoms — including from their doctors. “More than…
Treatment with platelets stripped of their natural sugar molecule coating helped to prevent inhibitors, or neutralizing antibodies, from forming against clotting factor VIII (FVIII) replacement therapy in a mouse model of hemophilia A, a study reported. Platelets promote blood clotting, and they are generally thought to boost inflammation…
Impairments in joint health are associated with certain functional deficits in the lower limbs among people with mild hemophilia, a study reports. A combination of aging, pain, and joint damage had a significant influence on patients’ performance in tests of muscle power, while aging alone explained most variation in…
Preventive treatment with Eloctate (efmoroctocog alfa) for up to five years led to sustained reductions in all bleed types for children, adolescents, and adults with severe hemophilia A, according to data from two Phase 3 trials and their open-label extension study. New analyses of these pivotal trials also…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…
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