When you live with a bleeding disorder, you quickly learn that the words people use matter almost as much as the treatments themselves. I’ve been called a “carrier” and a “hemophiliac,” and told that my condition was “mild” or “moderate,” as if these words fully described the challenges…
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AMT-060 was a first-generation gene therapy for hemophilia B that was tested in clinical trials as a potential treatment for reducing the risk of bleeding episodes in patients.
When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, according to follow-up data from the Alta Phase 1/2 clinical trial. All five men given the highest dose…
A panel of multidisciplinary experts have developed recommendations for applying best practice guidelines in the planning and managing of surgery in hemophilia patients. The guidelines were outlined in the consensus review, “Recommendations on multidisciplinary management of elective surgery in people with haemophilia,” published in the journal…
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…
Understanding hemophilia treatment options if you have inhibitors
Fact-Checked By Lindsey Shapiro, PhD
Hemophilia inhibitors can reduce treatment effectiveness and raise bleeding risks, but several therapies can help maintain control. Understanding your options can support more confident, informed care decisions.
In Brazil, hemophilia markedly impairs patients’ quality of life and ability to work, with up to 82.6% of people with the disorder being unemployed, a review study finds. Barriers to treatment include long distances to centers, lack of coordination of specialized and emergency care, and restricted access to rehabilitation.
In the past, no one believed women could have hemophilia. The medical community assumed that one X chromosome would compensate for the other. Doctors now know about the process of duplicate X chromosome inactivation, called “lyonization.” As the inactivation process is random, some female…
The other day, I picked up “MacDonald the Younger” from hemophilia camp. I enjoyed the trip through the mountains. The air felt fresh as I grew anxious to see my son. I wanted to hear all about his camp experience. What friends did he see? Did he try the…
Metagenomi is now working on preclinical studies to advance MGX-001, its experimental gene-editing therapy for hemophilia A, with the goal of bringing the treatment candidate into clinical trials in 2026. As part of a corporate update also noting 2024 progress, Metagenomi announced that, this year, it will be…