Long-term treatment with Esperoct (turoctocog alfa pegol) prevented bleeding in one-fifth of children with severe hemophilia A over the course of a Phase 3 clinical trial, according to final results from the study. Treatment was effective in both spontaneous and traumatic bleeds, and appeared to be…

Atomwise, a company that specializes in using artificial intelligence (AI) to develop small molecule medicines, will partner with GC Pharma to discover new ways of treating people with hemophilia. …

You are not alone. Coping with hemophilia as a patient, caretaker, or medical provider can seem overwhelming at times. When I am overwhelmed, have a question, or just want to talk to someone who I know will “get it,” I reach out to my “blood sisters” and…

Mononine, a long-standing treatment to prevent and control bleeding in hemophilia B, will be discontinued due to declining demand in recent years. CSL Behring, the therapy’s manufacturer, said the decision was based on the emergence of next-generation hemophilia B treatments now in greater use. “Over time,…

From early on, we find success by developing a strong work ethic. Some of us come by it naturally, while others learn the value of working hard and doing the best job they can do. I realized that discipline brought great rewards, both musically and academically. When I failed to…

Daratumumab — a cancer therapy marketed as Darzalex — can reduce levels of factor VIII (FVIII) inhibitors in people with hemophilia type A, researchers say. Their findings were published in a letter to the editor, titled “Daratumumab rapidly reduces high‐titre factor VIII inhibitors in…

Sometimes I am overwhelmed when I think about the hemophilia journey my sons are traveling. My first memories about this date to June 1996, when my first son, Julian, was born and diagnosed with severe hemophilia A. The early days of learning how to access a port, and later,…

A single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity over one month in men with severe hemophilia A, lasting longer in the body than the replacement therapy Advate, results from the EXTEN-A clinical trial suggest. Gains in half-life seen with BIVV001 “could signal…

The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…

When I have the opportunity to gather with my brothers and sisters from the hemophilia community, I notice we all have stories to tell. When you live with a rare disease, you are bound to have unique interactions with medical providers, family members, friends, and colleagues. These interactions range from…