Andreas Tiede: Treating our First Hemophilia B Patient with Gene Therapy
Andreas Tiede, Professor in the Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation at Hannover Medical School, eposted from Medizinische Hochschule Hannover on LinkedIn:
”A very special moment to share. Treating our first hemophilia B patient with gene therapy was a milestone for our team — and it’s amazing to see how much it has changed his life. Full of energy and plans for the future… what a difference a single infusion can make!”
Quoting Medizinische Hochschule Hannover‘s post:
”For the first time in Germany: MHH treats hemophilia B with gene therapy
Going on vacation, doing sports and feeling healthy – what is a matter of course for most people was only a dream for Marvin M. for a long time. The young man suffered from severe hemophilia B, a form of the so-called hemophilia, which severely restricted his life. The turning point came with a new type of treatment at the Clinic for Hematology, Hemostaseology, Oncology and Stem Cell Transplantation at the Hannover Medical School (MHH). Marvin M. received gene therapy and now has a great chance of good health in the long term. He is the first patient in Germany to be treated with this type of therapy for hemophilia B.
Hemophilia B is an inherited disorder of blood clotting that is due to the absence of clotting factor IX. The symptoms are the same as with hemophilia A. “In those affected, the blood plasma clots more slowly. There is a risk that the smallest injuries or even spontaneously lead to bleeding into large joints and muscles, sometimes even into the gastrointestinal tract or the central nervous system,” explains Professor Dr. Andreas Tiede from the Department of Hematology, Hemostaseology, Oncology and Stem Cell Transplantation. The bleeding has serious consequences. As a result, joints can be completely destroyed in the long term, for example. Both men and women are affected by the hereditary disease. However, it is almost exclusively men who have severe characteristics. Professor Tiede estimates the total number of severe hemophilia B patients in Germany at around 500. In them, the activity of coagulation factor IX is less than two percent of the norm. To increase clotting activity, they have to inject themselves with the missing factor IX for a lifetime. Since this has a short half-life, twice a week by default.”
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