by Saarland University Credit: CC0 Public Domain For people with the bleeding disorder hemophilia, life can seem like one continuous state of alertness. If they accidentally cut their finger while chopping vegetables, they have to immediately administer the clotting factor protein that their body either lacks or does not produce enough of. A normal visit to...
Tag: <span>hemophilia</span>
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Hemophilia A Gene Therapy Durable at 2 Years; Under FDA Review
M. Alexander Otto, PA, MMS February 22, 2023 Clinical data have been published for a gene therapy for hemophilia A that is approaching the market — valoctocogene roxaparvovec (Roctavian), which is currently under review by the US Food and Drug Administration. Hemophilia A (a deficiency of clotting Factor X) is the most common form of the disease, accounting for...
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FDA approves CSL’s gene therapy for hemophilia
November 22, 2022 2:56 PM MST Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo Nov 22 (Reuters) – A one-time gene therapy for hemophilia B from Australian drugmaker CSL Ltd (CSL.AX) has been approved by the U.S. Food and Drug Administration, potentially...
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Gene therapy for hemophilia A shows promise in phase 3 clinical trial
by Bob Yirka, Medical Xpress Graphical abstract. Credit: New England Journal of Medicine (2022). DOI: 10.1056/NEJMoa2113708 An international team of researchers has found more evidence that a new gene therapy may help some people with hemophilia A. In their paper published in the New England Journal of Medicine, the group describes the characteristics of the clinical trial and...