Newsroom
In our Newsroom we share some recent headlines and links to articles that may be of interest to our bleeding and clotting disorder patients, families, and providers.
NBDF Recommends Routine Iron Screening for People with Bleeding Disorders
June 16, 2026
The National Bleeding Disorders Foundation has released standardized guidelines for screening for iron deficiency for people who have bleeding disorders, and the recommendation should help give doctors clear guidance on when to screen.
FDA Expands Hympavzi to Include Pediatric Treatment
June 9, 2026
The FDA approved an expanded indication for Hympavzi (marstacimab), allowing it to be used in children ages 6-11 years with hemophilia B, with or without inhibitors. The expanded approval allows the treatment to become the first non-factor therapy available for pediatric patients in this age group.
Trial Shows Breakthrough Bleeds and Minor Surgeries Successfully Treated for Patients on Hympavzi
May 19, 2026
A large clinical trial showed that the use of prophylactic injection therapy of Hympavzi (marstacimab) for adults and children helped prevent bleeding episodes for patients with both hemophilia A and B. Breakthrough bleeds that did occur, or minor surgical procedures that were needed were effectively treated with standard clotting factor replacement therapy.
FDA Grants Fast Track Status to 1-Time Stem Cell Therapy for Hemophilia A
May 5, 2026
The FDA has awarded both fast track and rare pediatric disease designations to a single-use stem cell therapy being developed for hemophilia A by Expression Therapeutics. The decision follows a Phase 1 clinical trial showing a single administration of the therapy led to sustained factor expression and the annual bleeding rate dropped to zero.
New Study Finds Bispecific Antibody Prophylaxis Reduces Bleeding in Hemophilia A
May 4, 2026
Researchers in Italy have found that prophylaxis with Mim8, a novel bispecific antibody designed to replicate function of activated factor VIII, was superior to both on-demand therapy and clotting factor concentrate prophylaxis in reducing annualized rate of bleeding events, regardless of inhibitor status.
Research Shows Regular Excercise Eases Chronic Pain in Hemophilia
April 17, 2026
A study published in Pain Management Nursing reviewed published research about the effect of physical excercise, including aerobic, strength training, and balance exercises, found that regular exercise may help ease chronic pain for those with hemophilia.
Study Finds Protein Marker May Detect Early Joint Damage for Hemophilia A Patients
April 3, 2026
A study in Greece compared the YKL-40 protein levels - a protein associated with inflammation - in a small group of boys and men with severe hemophilia A and found that those with known joint damage had signficantly higher levels. The findings offer a potential new way to monitor joint damage in bleeding disorders patients.
Survey Shows Little Interest in Gene Therapy for Many Hemophilia Patients
March 20, 2026
A small survey in Germany found that many people with severe hemophilia are not interested in receiving gene therapy. Researchers found that despite having only a general knowledge of the treatment option, more than half of those surveyed had a predominantly rejective attitude toward the therapy.
CSL Behring Announces Temporary Disruption in Hemgenix Supply
March 18, 2026
CSL Behring announced that its hemophilia B gene therapy, Hemgenix, was experiencing a temporary global stockout of the treatment. The company shared that the supply issue was not related to safety or effectiveness of the medication, but reflects the complexity of manufacturing gene therapies while maintaining regulatory and quality standards.
Study Shows Lower-Dose Gene Therapy Effective in Hemophilia A
March 17, 2026
A Phase 1 pilot study in China showed their experimental gene therapy GS001 safely increased the levels of factor VIII for hemophilia A patients for almost three years. The treatment reduced bleeds and the need for replacement therapy.
Gene Therapy for Hemophilia B Continues to Show Factor Production after Six Years
March 10, 2026
A single infusion of Beqvez, a now-discontinued gene therapy by Pfizer for hemophilia B, led to sustained production of factor IX for up to six years. The treatment reduced bleeding episodes and eliminated the need for routine preventative infusions in most patients. The study was published in Blood Advances.
BioMarin Pulls Gene Therapy Roctavian from US Market
February 26, 2026
BioMarin Pharmaceuticals has announced it is pulling the hemophilia A gene therapy Roctavian from the US Market. The move followed an announcement in October 2025 that it was looking for a buyer for the expensive therapy. The treatment will remain available to the US community through the end of May 2026.
News Content Disclaimer:
Links to articles listed may take readers to websites separate and out of the control of the CU School of Medicine and the Hemophilia and Thrombosis Center. The CU School of Medicine and the Hemophilia and Thrombosis Center are not responsible for any errors, inaccuracies or opinions that may be expressed in any of the linked articles.
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