Weekly treatment with the experimental therapy marstacimab (PF-06741086) was found to significantly reduce bleed rates for adult and adolescent hemophilia A and B patients without inhibitors relative to standard therapies. In so doing, it met the main goal of the pivotal Phase 3 BASIS clinical trial, designed to…
A 48-year-old man with severe hemophilia A developed a rare intramural hematoma with blood leaking into one of the layers of the intestine wall that was successfully treated with an early infusion of the missing blood clotting factor, a recent case report described. Prophylactic treatment prevented its recurrence. A…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to SerpinPC, Centessa Pharmaceuticals’ investigational treatment for people with hemophilia B. Fast track status expedites the review of therapy candidates that show a potential to fill an unmet medical need for serious conditions. It also allows more…
Most people with hemophilia responding to a survey in the Netherlands were generally satisfied with their treatment care. Patients’ interactions with healthcare professionals, availability of care, and coordination were cited as the most important factors for satisfaction. However, they reported improvements were needed regarding information about new treatments, their…
People with hemophilia showed no heightened protection against COVID-19 severity or mortality, a large retrospective U.S. outcomes study found. “Having hemophilia was not a risk factor for death,” the researchers wrote, noting, however, that hemophilia patients “were more likely to have moderate COVID-19 severity and to be hospitalized.” Older…
Hemophilia treatments in Thailand are limited due to a lack of budgetary resources, the high cost of medications, and an infrastructure inadequate for supporting patient care, a study found. These hurdles mean that people with hemophilia in the Southeast Asian country may not be able to access the care…
Female hemophilia AÂ patients and carriers have more frequent bleeding than males, yet a much smaller proportion receive replacement therapy to prevent or stop them, a U.S. study finds. The medical and pharmacy costs for women with hemophilia A or carriers were up to about 14 times lower…
Note: This story was updated May 8, 2023, to remove hemophilia as one of the specific indications the collaboration will focus on, as these have not yet been publicly disclosed. AstraZeneca and Sernova are teaming up for a preclinical research project that aims to develop new cell therapies…
2seventy bio announced that it has achieved a key preclinical milestone under its joint program with Novo Nordisk to develop a gene-editing tool to treat hemophilia A. Details of the “proof of concept” research that met this milestone — reported to be worth $15 million to the…
Patient dosing has been completed in a registrational Phase 3 clinical trial in China testing BBM-H901, Belief BioMed‘s (BBM)’s investigational gene therapy for hemophilia B, the company announced. BBM-H901 is designed to restore the production of factor IX (FIX) — the blood-clotting protein that is faulty or missing…
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