Hemophilia C is a rare bleeding disorder that affects the blood’s ability to clot.
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Sangamo Therapeutics has dosed the first patient in a Phase 1/2 clinical trial testing its investigational genome-editing therapy, SB-FIX, for patients with severe hemophilia B. The Georgetown University and MedStar Georgetown University Hospital are conducting the study, which is still recruiting participants in nine sites across the United…
The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to SHP654 (BAX 888), an investigational factor VIII (FVIII) gene therapy candidate being developed by Shire for the treatment of hemophilia A. Gene therapy allows the delivery of a functional copy of the defective gene.
Last week, I read Gill Paul’s latest novel, “The Lost Daughter.” The book is a historical fiction account of Russia’s Romanov family. It is well-known that the Romanovs and hemophilia are connected, but I had no expectation that bleeding would play into the plot. Near the beginning of…
The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for eligible adults with severe hemophilia A. Roctavian now becomes the first gene therapy to win FDA approval for the treatment of hemophilia A in the U.S. “Today’s approval of Roctavian builds…
When given as a prophylactic, or preventive therapy, or on an on-demand basis, FEIBA (factor eight inhibitor bypassing activity) was deemed safe and effective at lessening the number of bleeds in people with severe hemophilia and high levels of inhibitors. That’s according to four-year data from a real-world…
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Gene Therapy Approach, Tested on Liver Cells and Mice, Seen as Potential Treatment for Hemophilia B
Researchers, working with mice, have developed a new way of producing substantial numbers of gene-corrected liver cells with the possibility of repopulation in vivo. The strategy, which relies on the liver’s unusual regenerative skills, may hold promise for the treatment of diseases such as hemophilia B. The study by scientists at Oregon Health & Science University,…
The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…
NovoSeven (eptacog alfa [activated]) is a bypassing agent that’s approved to treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A or B who have developed inhibitors — neutralizing antibodies against certain clotting factors.
Hemophilia can affect many aspects of life, including sexual health. If you have hemophilia, sexual activity is generally safe as long as certain precautions are taken.