Patricia Inácio, PhD,  science writer—

Two new patients in the U.S. have received Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B, per new reports. The therapy was first administered last year in May to a patient at UC San Diego Health, per a University of California, San Diego, news…

The first-in-human Phase 1/2 clinical trial testing BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B, is now enrolling patients at two sites in the U.S. BE-101, a first-in-class therapy, aims to address the persistent unmet needs of hemophilia B patients — particularly the burden of ongoing treatment…

Sevenfact (eptacog beta) was deemed safe and effective at stopping bleeds in patients with hemophilia A or B with inhibitors, according to real-world data from a case series study in the U.S. The treatment was also more affordable than other similar therapies and effective even in patients who’d failed…

Hemlibra (emicizumab) effectively prevented bleeds in patients with acquired hemophilia A (AHA), according to real-world data from a single center study in Japan. The treatment also led to shorter hospital stays, helped patients maintain their daily living activities, and contributed to lower the doses of bypassing agents,…

Preventive therapy, or prophylaxis, in patients ages 50 and older with severe hemophilia type A  and type B was effective at reducing bleeds, including those in target joints, and improving some aspects of their quality of life. That’s according to findings from a post hoc analysis of four…

A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B. The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding…

Hemgenix (etranacogene dezaparvovec), the first approved hemophilia B gene therapy, has been successfully administered to two patients in France, marking the first time the treatment has been given in a real-world setting in Europe. The gene therapy, originally developed by uniQure and marketed by CSL Behring, was…

Most caregivers or patients with hemophilia who were on preventive treatment were satisfied or very satisfied with the Florio Haemo smartphone app, even after prolonged use, a survey study suggests. The app lets patients track their infusions, bleeds, pain, and activities, and helps them manage their medication stock.

The efficacy and safety profiles of preventive treatment with Hemlibra (emicizumab-KXWH) are not affected by age or the presence of some simultaneous health conditions, that is, comorbidities, in older people with hemophilia A. The findings are the result of a post hoc analysis of pooled data from Phase…

In Brazil, hemophilia markedly impairs patients’ quality of life and ability to work, with up to 82.6% of people with the disorder being unemployed, a review study finds. Barriers to treatment include long distances to centers, lack of coordination of specialized and emergency care, and restricted access to rehabilitation.