The European Commission has granted conditional marketing authorization to BioMarin’s one-time gene therapy Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe hemophilia A who have neither inhibitors nor detectable antibodies against adeno-associated virus serotype 5 (AAV5). The decision, which comes a couple of months after a…
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Long-term prophylaxis or preventive treatment with Eloctate (efmoroctocog alfa) helps to ease pain and improve health-related life quality for adults and children with hemophilia A, according to an analysis of three Phase 3 clinical trials. “These analyses highlight the relevance of pain, physical health, and overall wellbeing as…
It’s easy to look back and think about what one could have done differently. Handling a crisis with grace rather than anger, calling the doctor immediately instead of waiting, or keeping a diagnosis secret to avoid others’ judgment. We all make mistakes and, hopefully, we learn from them. Forgetting…
In February, I wrote about hemophilia’s effects on bone health after attending a webinar sponsored by Factor My Way, a patient support program. It featured bleeding disorders expert Claudio Sandoval, MD, who explained that everyone with hemophilia A (factor VIII deficiency) is at an increased risk of…
The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic liver-specific promoter to the cell’s nucleus by using recombinant adeno-associated virus (rAAV). The…
Preventive treatment with Altuviiio (efanesoctocog alfa) provided better bleed prevention than Hemlibra (emicizumab) in adults and adolescents with hemophilia A, according to an indirect comparison of Phase 3 clinical trial data. “While it should be borne in mind that this is an indirect comparison and, as such,…
As a parent, it is my responsibility to help my sons become productive, compassionate, caring individuals. My oldest son, Julian, is 25 and working to complete his college degree. He is on the cusp of transitioning to a new phase in life. In less than a year, he will…
From the time my wife found out she was pregnant, we celebrated a ritual each evening until the birth of my oldest son, Julian. Before we kissed good night, I put my face to her belly and sang the song “For Baby (For Bobbie)” to him. Without seeing…
Antifibrinolytics are medicines that promote blood clotting by preventing or slowing down a process called fibrinolysis, which is the break down of blood clots. Antifibrinolytics are used as a treatment for hemophilia, in surgical procedures to prevent excessive blood loss, and for heavy menstrual bleeding. How antifibrinolytics work…
Hemophilia is an inherited disease that prevents blood from clotting, a process that’s required to stop bleeding. According to the Hemophilia Federation of America, there are various complications that can arise from having a bleeding disorder like hemophilia. MORE: 4 Hemophilia Screening Tests Joint damage: One of the most frequent complications…