When I was a young woman, I was very negative. I would look ahead to a situation and immediately imagine the possible pitfalls. I never considered everything would work according to plan. When plans went better than expected, it was a bonus. As a former glass-half-empty kind of person,…
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European regulators have granted orphan drug status to CB 2679d, a hemophilia B therapy that Catalyst Biosciences developed to prevent bleeding episodes. The therapy, known as ISU304 in South Korea, prevents bleeding by increasing hemophilia B patients’ level of a blood protein called Factor IX, which is deficient in the disease.
Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy approved for hemophilia B, has been approved in Switzerland, CSL Behring, the company marketing the therapy globally, announced. “The approval of Hemgenix in Switzerland marks an important milestone for patients, and we look forward to collaborating with…
The World Federation of Hemophilia (WFH) is commemorating having successfully distributed, since 1996, over one billion international units (IUs) of factor and non-factor replacement therapies for people with inherited bleeding disorders, such as hemophilia, through its Humanitarian Aid Program. Donations from the program have improved the quality…
Pfizer Rare Disease has developed a new video game and wearable wristband to help hemophilia patients learn more about their condition and promote activity tracking in the U.S. The video game, Hemocraft, and the HemMobile Striiv Wearable wristband were launched at the National Hemophilia Foundation’s 69th Annual Meeting in Chicago, Aug. 24-26.
Last updated May 29, 2024, by Lindsey Shapiro, PhD Â Fact-checked by Joana Carvalho, PhD What is Kogenate FS for hemophilia? Kogenate (octocog alfa) was an approved treatment used to prevent bleeding episodes, including those occurring during surgery, in people with hemophilia A that has now…
A single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained increases in the activity of factor IX (FIX), effectively preventing bleeds and the need for replacement therapies in people with severe hemophilia B, a Phase 1/2 clinical trial shows. These results support the…
My sons have severe hemophilia A and are 10 years apart. Such a considerable age difference once posed challenges, especially when they were younger. Back then, my oldest son, Julian, felt he and his brother, Caeleb, didn’t have common interests. I reminded Julian that siblings close in age…
SerpinPC, an experimental treatment being developed by Centessa Therapeutics, continues to be safe when used for up to nearly three years and to almost zero out the number of bleeds in adults with severe hemophilia A or B. Those findings come from a one-year extension of the…
uniQure, a company specializing in human gene therapy, will present clinical data from a Phase 1/2 dose-escalating study of AMT-060 in adults with hemophilia B at the upcoming 58th American Society of Hematology Annual Meeting (ASH 2016), to be held in San Diego, Calif., in Dec.3-6. The…