All of the hemophilia B patients in an ongoing Phase 1/2 clinical trial of SPK-9001 experienced clotting-factor increases and reductions in bleeding episodes, according to the therapy’s maker, Spark Therapeutics. University of Pennsylvania medical researcher Adam Cuker will present the updated preliminary results today at the Hemostasis and Thrombosis Research Society (HTRS) 2017…

Note: This story was updated Oct. 15, 2024, to note results from the subset of patients with inhibitors participating in BASIS are now expected next year. A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for…

The investigational medication marzeptacog alfa activated (MarzAA) showed promise at stopping bleeding in animal models of hemophilia. The findings were presented in two posters at the Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) by researchers from Catalyst Biosciences, which is developing MarzAA. MarzAA is…

My adult son, Julian, does a wonderful job managing his hemophilia. He infuses twice a week per doctor’s orders, and if he has a breakthrough bleed, he treats as needed. Julian is extremely fortunate, because throughout his 24 years, hemophilia has not been the center of his life. His journey…

Novo Nordisk expects a decision this month by the U.S. Food and Drug Administration (FDA) on its product N9-GP  (nonacog beta pegol) as a treatment for hemophilia B. Approval of the drug may depend on the conclusions the FDA makes regarding safety concerns raised by its blood products advisory committee,…

Our baby girl turned one earlier this year. Watching Cittie mature makes me reflect on the innocent phase of childhood. Life was so simple back then. I believed games and toys to be major issues of the utmost importance. I stayed late after school and played with my friends.

With the holiday season in full swing, it’s the perfect opportunity to take a look back at 2017. It’s been a year full of ups and downs, and while there were definitely quite a few low moments, there were certainly quite a few highs. To celebrate the year that was,…

Prophylaxis with Idelvion (rIX‐FP) significantly improved clinical outcomes and quality of life for children with hemophilia B and eased daily life for their caregivers, Phase 3 clinical trial data show. The findings of the study, “Health-related quality of life in paediatric haemophilia B patients treated with…

Greater awareness of hemophilia and its symptoms is associated with better adherence to treatment by patients, and those with more severe disease are most likely to use a treatment as prescribed, a study based on a new questionnaire shows. Responses were seen to validate this questionnaire, which addresses…