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…
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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…
Fresh off the heels of the National Hemophilia Foundation’s virtual symposium earlier this month, I just attended another online conference hosted by the Hemophilia Federation of America. One thing that drew me to this event was the number of sessions dealing with mental health, for both caregivers and…
Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…
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…
The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In a complete response letter that took the therapy’s developer, BioMarin by surprise, the FDA said it needed…