Two new patients in the U.S. have received Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B, per new reports. The therapy was first administered last year in May to a patient at UC San Diego Health, per a University of California, San Diego, news…
I have to admit, I was captivated by Hemophilia News Today‘s Facebook page during the final days of December, eagerly following its countdown of the year’s top 10 most-read columns. It was an incredible honor to see that my columns secured eight of those 10 spots. Each one…
The start of a new year often brings a surge of enthusiasm, hope, and resolutions. We might set ambitious goals like, “This is the year I lose weight.” Or, “This is the year I go to the gym five days a week.” Or, “This is the year I mend a…
Alhemo (concizumab-mtci) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with inhibitors. It is given as daily under-the-skin, or subcutaneous, injections that can be self-administered.
Me: “Hello?” Medical assistant: “Hi, Shellye, the doctor wants to see you ASAP to discuss your recent test results. She’s concerned.” Me: “Oh no, that doesn’t sound good. Can you tell me what’s going on?” Medical assistant: “We just got your baseline bone density tests back and they show you…
Sangamo Therapeutics will regain full rights to develop and market giroctocogene fitelparvovec, a gene therapy candidate for adults with moderately severe to severe hemophilia A, after Pfizer decided to end its involvement in the program. Pfizer’s decision not to advance into regulatory submissions came despite positive…
The U.S. Food and Drug Administration (FDA) has approved Novo Nordisk’s concizumab, which will be sold under the brand name Alhemo, as a daily treatment to prevent or reduce the frequency of bleeding episodes in certain adults and adolescents with hemophilia. Eligible patients are those with hemophilia…
In 2024, Hemophilia News Today provided readers with up-to-date coverage on research, treatments, and clinical trials for hemophilia. We compiled the top 10 most-read stories of the year, each with a brief summary. As we enter 2025, we look forward to continuing to support the hemophilia community. We…
Two and a half years ago, I purchased a manufactured home in need of a few repairs. Truthfully, it had really good bones, but was a dump. But I saw the potential in it and knew that with enough love, time, and renovation it would be a beautiful place to…
December not only marks the end of the year, but it’s also the time for my annual visit to the specialists at my regional hemophilia treatment center (HTC). I live more than two hours away from them, so I appreciate the convenience of telehealth appointments, which are far more…
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