MeSH tree: C15.378.100.100
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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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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Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him.
Jun 21, 2024
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Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting disorder, the company said Friday.
Apr 26, 2024
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Researchers at the Wake Forest Institute for Regenerative Medicine (WFIRM) have recently published an article in Nature Communications that demonstrates the potential of bioengineered human placental cells as a cure for Hemophilia ...
Nov 30, 2023
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After a bump, we can expect a bruise. But what if we find ourselves bruising without any noticeable cause? What might be behind it? Should we worry?
Oct 20, 2023
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