After five years of follow-up, Hemgenix (etranacogene dezaparvovec-drlb) was associated with durable factor IX (FIX) levels, lower bleeding rates, and a substantial reduction in the need for regular FIX infusions, with a generally favorable long-term safety profile in men with severe or moderately severe hemophilia B. These data…
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In 2025, Hemophilia News Today delivered readers timely reporting on research, therapeutic options, and clinical trials for hemophilia. Below is a list of the top five most-read stories we published this year. As we enter 2026, we look forward to continuing to support and serve as a source…
I have a bad habit. When I shop for groceries, I grab what I need but often get distracted by a bright package. A new cookie? How many carbs are in it? The same thing happens when I watch a movie or listen to a podcast with lots of twists.
BBM-H901 (dalnacogene ponparvovec), a gene therapy that’s approved in China for adults with moderate to severe hemophilia B, was shown to significantly reduce bleeding rates among hemophilia B patients in clinical trials, a new study shows. Hemophilia B is a genetic disorder caused by mutations in the gene that…
Planning a pregnancy with hemophilia can bring up a lot of questions. While many pregnant women with the condition don’t experience major complications, careful planning and comprehensive care are needed to ensure the best outcomes for you and your baby. Learning about hemophilia and pregnancy, including…
Hympavzi (marstacimab) reduced bleeding rates and improved quality of life in adults and adolescents with hemophilia A or B who have inhibitors (antibodies against standard replacement therapies). That’s according to final data from the Phase 3 BASIS study (NCT03938792), which demonstrated the treatment met its main goal of…
My husband, Joe, and my sons, Julian and Caeleb, each have their own unofficial jobs when we decorate for the holidays. Julian brings the dusty red and green totes full of Christmas decorations in from the shed. I open them and decide what comes inside first. Joe and Caeleb…
Julian MacDonald opens up about life with severe hemophilia A, from early diagnosis to the financial and insurance challenges he faced in adulthood. He shares how leaning on pharmacists, care teams, and the broader hemophilia community became essential to understanding he didn’t have…
When people hear the word “hemophilia,” they usually picture bleeds, factor infusions and other treatment routines, and trips to the hospital. Those things certainly shape the condition, but they’re not the whole story. Over the years, I’ve noticed something that feels harder to explain, yet…
A newborn in China was diagnosed with hemophilia B after a ruptured liver caused significant abdominal bleeding, according to a case report. Due to early detection, timely surgical repair, and immediate factor-replacement therapy, the infant made a full recovery. This case shows that “neonatal hepatic rupture may be…