MeSH tree: C15.378.100
For U.S. patients with severe hemophilia A, prophylaxis with efanesoctocog alfa is neither conventionally nor distributionally cost-effective, according to a study published online April 22 in the Annals of Internal Medicine.
Apr 28, 2025
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The U.S. Food and Drug Administration has approved Sanofi's Qfitlia (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with ...
Apr 3, 2025
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Scientists have taken an important step toward the development of a potential new treatment for treating Shiga toxin-producing E. coli (STEC), the notorious bug associated with severe food poisoning.
Apr 1, 2025
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As scientists, we often think we understand a virus—its structure, its tricks, the way it moves through the body. But every once in a while, we stumble upon something unexpected—something that completely changes the way we ...
A recent study led by MedUni Vienna provides new insights into the mechanisms of coagulation in people with hemophilia A, the most common form of hemophilia. The research team was able to show that saliva contains special ...
Jan 14, 2025
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Two researchers from Huntsman Cancer Institute at the University of Utah (the U) have discovered a gene variant found in high-altitude Andean populations that they believe could be a new biomarker for predicting the severity ...
Dec 13, 2024
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Research led by Christian Medical College in Vellore, India, has demonstrated the successful use of lentiviral vectors to deliver gene therapy for patients with severe hemophilia A. The study presents a potential alternative ...
RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
The U.S. Food and Drug Administration (FDA) approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with ...
Oct 15, 2024
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Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71% after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published in the New ...
Sep 25, 2024
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