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Gene Therapy a Boon for 6 Hemophilia Patients
Posted 12 Dec 2011 by Drugs.com
SATURDAY, Dec. 10 – A single treatment of gene therapy dramatically improved symptoms and quality of life in a small group of men with hemophilia B, an uncommon form of the bleeding disorder, a new study suggests. "It is a small trial in that only six patients have been treated thus far, but it has been rather successful and I think it will encourage further use of this approach in the very near future, both by us and others," said senior author Dr. Andrew Davidoff, chairman of the department of surgery at St. Jude Children's Research Hospital in Memphis. "At this point, we're a little shy of being able to call it a cure, but it's a good start." Six severely affected patients were infused with a correct version of the gene that makes a protein called factor IX, which is essential for blood clotting. Following treatment, four were able to stop receiving protein injections to prevent ... Read more
Related support groups: Hemophilia A, Hemophilia B
Using Drug for Prevention Might Help in Hard-to-Treat Hemophilia
Posted 2 Nov 2011 by Drugs.com
WEDNESDAY, Nov. 2 – A compound currently used to stop bleeding episodes in a subset of hard-to-treat hemophilia patients also seems to be effective in preventing bleeds before they start. Although this study, published in the Nov. 3 edition of the New England Journal of Medicine, was a small one and didn't last long, the results do raise the possibility that, over time, this type of use will also prevent the much-feared joint damage that is a hallmark of the condition. "This study shows that it is at least possible to keep these people relatively healthy, without bleeds, by using chronic prophylactic [preventive] agents," said Dr. Thomas Harrington, director of the Adult Hemophilia and Adult Sickle Cell programs at the University of Miami Miller School of Medicine. He was not involved with the study. The research was funded by Baxter BioScience, which makes FEIBA (Factor VIII Inhibitor ... Read more
Related support groups: Hemophilia A, Hemophilia B
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Coagulation Defects and Disorders
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Novoseven, Konyne 80, BeneFIX 250 Int'l Units, Profilnine SD, Alphanine SD, Bebulin VH, NovoSeven RT, Mononine, coagulation factor viia, factor ix complex, BeneFix, Proplex T, coagulation factor ix