When my son “MacDonald the Older” took over his care, I celebrated his accomplishment. “MacDonald the Younger” would not reach independence until many years after his older brother did, but reducing oversight of infusing to one child proved liberating. Before the Younger’s independence, my wife and I still actively participated…
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Hemophilia is best known for symptoms such as prolonged bleeding after an injury, easy bruising, and spontaneous bleeding into joints or muscles. Over time, repeated bleeding can also lead to less common complications. One of the rarest is the development of hemophilic pseudotumors, or benign masses caused by…
The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal — which…
Raising two sons with a bleeding disorder has given me hundreds of stories. Many of them lead to happiness and gratitude. Others are filled with pain and suffering. They are all a part of me in a way I never imagined they would be. The nice thing about having so…
Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) for treatment of adolescents and adults with hemophilia B. The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for…
TU7710, an experimental treatment for managing bleeds in people with hemophilia who have inhibitors, demonstrated an average half-life that ranged from 10.4 to 16.6 hours across different doses in a Phase 1a study with healthy volunteers. This extended half-life, that is, the time it takes for a medication’s levels…
Prophylactic treatment for hemophilia can help people with the disease avoid unemployment due to disability, a study reports. Hemophilia has a negative effect on a person’s employability. A 2022 global report by the World Federation of Hemophilia indicates about 18% of hemophilia patients see their employment status impacted…
Treatment with Idelvion (rIX-FP) helps children and adults with hemophilia B stick to prophylactic dosing schedules by reducing treatment burden, according to a study using clinical trial and real-world data. The study, “High adherence to prophylaxis regimens in haemophilia B patients receiving rIX‐FP: Evidence from clinical…
In recognition of World Hemophilia Day (WHD), Catalyst Biosciences is hosting an “Ask Me Anything” (AMA) session on Reddit on April 17. During the Reddit AMA event, which will take place at noon EST Wednesday, clinicians will answer questions from patients and caregivers about bleeding disorders including hemophilia. A…