Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then move in pivotal clinical trials of SerpinPC — an investigational therapy for hemophilia A and B patients, regardless of disease severity or inhibitor status — that it plans to launch…
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I had a lot to learn about hemophilia when I attended my first retreat for women with bleeding disorders in 2017. Some of the attendees were comparing their levels of clotting factor VIII (FVIII), the protein that’s deficient in hemophilia A, when I was shocked to learn mine were the lowest…
Sports and physical activity can benefit people living with hemophilia, according to a recent study. Researchers in Germany and the United Kingdom investigated to find whether participating in sports can benefit adults with varying degrees of hemophilia severity. The report, “The impact of sport on health status,…
The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others in the hemophilia community by surprise. That decision, announced in a complete response letter of…
Biomarin Pharmaceutical is looking to sell off rights to Roctavian (valoctocogene roxaparvovec-rvox), its approved gene therapy for hemophilia A. “As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest Roctavian and remove it from…
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BioMarin’s Gene Therapy for Hemophilia A Continues to Prevent Bleeds After Four Years, Trial Shows
BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…
BrightInsight has launched a web-based dosing calculator to help physicians select the correct loading and maintenance dose of the treatment Hemlibra according to the body measurements of their patients with hemophilia A. The dosing calculator, launched with the support of Hemlibra’s manufacturer, Roche, will support the…
In an interview with Genentech, 13-year-old Beau talks about what it’s like living with the bleeding disorder hemophilia. MORE: Seven recommended books to learn more about hemophilia Beau is just like other boys his age — in his spare time he plays computer games and is…
CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer, for the first time successfully treated a patient with acquired hemophilia A (AHA), a study reported. The study, “Anti-CD19 CAR-T cell therapy for acquired hemophilia A,”…