One of the stranger skills I’ve developed in my marriage to someone with hemophilia isn’t medical — it’s domestic. It’s the art of removing blood stains. To be fair, I already had some experience dealing with blood simply by being a female person who menstruates. But when you’re married…
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I am a musician who has played in orchestra pits, wind ensembles, chamber groups, and orchestras. I even played antiphonal oboe one Christmas! Music is powerful. It transforms you and allows you to escape for a few hours from the regular happenings of the day. However, most people…
CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, can increase factor IX (FIX) levels and significantly reduce bleeding, early studies show. The data, “Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B…
A committee of the European Medicines Agency (EMA) has issued a positive opinion recommending the one-time gene therapy etranacogene dezaparvovec be granted conditional marketing authorization to treat appropriate adults with hemophilia B. The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will now be…
Obizur (susoctocog alfa), a pig-derived replacement therapy, can be used to prevent bleeding in people with acquired hemophilia A who need to undergo surgery, a new case report highlights. The report, “Successful perioperative prophylaxis with susoctocog alfa in a patient with acquired haemophilia A: A…
The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia B, making it the first approved gene therapy for the disorder in the country. BBM-H901 was developed and will be manufactured by Belief Biomed (BBM), but it will be commercialized…
As I mentioned in an earlier column, the news that my firstborn son had hemophilia caught my wife and me entirely off guard. When we spoke with the obstetrician, the only word we heard was hemophilia. We later learned that my son had another diagnosis: a low inhibitor titer. The…
As I sat in my office chair working on bills and other household needs this past Saturday afternoon, my youngest son, Caeleb, approached me and asked if we could talk for a few minutes. His mannerisms told me he needed to share something dire, so I stopped typing, turned to…
UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The designations…
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…