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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
Kogenate FS Prevents Joint Damage in Young Hemophiliacs
Posted 10 Oct 2008 by Drugs.com
FRIDAY, Oct. 10 – The U.S. Food and Drug Administration has approved Kogenate FS to help reduce bleeding episodes and prevent joint damage in children with hemophilia A, the most severe form of the disease. Hemophilia occurs when a protein that's needed for blood to clot is either deficient or missing entirely. Kogenate FS is a genetically engineered version of the deficient protein, known as factor VIII. The product was first licensed by the FDA in 1993 to control bleeding during or after surgery. Hemophilia A, which occurs almost exclusively in males, affects about 15,000 people in the United States, the agency said in a news release. When hemophiliacs are injured, they bleed longer than people without the disorder. When bleeding occurs in the muscles and joints of hemophiliacs, they are at increased risk of joint damage. The drug was clinically tested in 65 boys under 30 months of ... Read more
Related support groups: Hemophilia A, Advate
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Hemophilia A with Inhibitors, Coagulation Defects and Disorders
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DDAVP, desmopressin, tranexamic acid, antihemophilic factor, Novoseven, Cyklokapron, Monarc-M, Koate-DVI, Biostate, view more... Factane, Kogenate Bayer, Recombinate, Hyate:C, Koate-HP, Monoclate, Helixate, Xyntha, NovoSeven RT, Stimate, Kogenate FS with Bioset, Hemofil-M, Bioclate, Helixate FS, Helixate NexGen, Kogenate, Monoclate-P, Advate, Humate-P, Autoplex T, Feiba VH Immuno, Kogenate FS, antihemophilic factor/von willebrand factor, coagulation factor viia, Genarc, Wilstart, Haemate P, anti-inhibitor coagulant complex, Replenate, Liberate, Fanhdi, Beriate P, Optivate, Alphanate, ReFacto