MeSH tree: C16.320.099.500
A study in China covering 42,703 families affected by rare diseases across 32 provincial regions of China has established a new diagnosis framework for rare diseases. It offers new hope to millions of patients struggling ...
Sep 2, 2025
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Scientists from Cincinnati Children's and colleagues based in Japan report achieving a major step forward in organoid technology: producing liver tissue that grows its own internal blood vessels.
Jun 25, 2025
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Researchers from the Centenary Institute have uncovered a reason why some people with hemophilia A develop resistance to their critical treatment, paving the way for the development of more effective therapies. The study ...
May 28, 2025
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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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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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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 ...
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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For children with severe hemophilia A, once-weekly prophylaxis with efanesoctocog alfa is associated with high sustained factor VIII activity, resulting in effective prevention of bleeding, according to a study published ...
Jul 19, 2024
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In a clinical practice guideline issued by the International Society on Thrombosis and Haemostasis and published online June 13 in the Journal of Thrombosis and Haemostasis, recommendations are presented for the treatment ...
Jul 1, 2024
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