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.
Small Study Finds Little Genetic Testing Done for Girls at Risk of Being a Hemophilia Carrier
May 9, 2025
A small study done at one US HTC found that relatively little genetic testing was done for girls who had a 50% risk of being a hemophilia carrier. The study indicated that there may be several barriers in place preventing genetic testing.
Scholarships Available for Bleeding Disorder Patients
April-June
Several scholarships are available to people with bleeding disorders and some are available to family members. These each have different qualifications and deadlines. Bleeding.org has a list of the details and deadline dates at their website.
Survey Finds Blood Group O Not Tied to Inhibitor Risk in Kids with Severe Hemophilia A
May 6, 2025
Conflicting study results led researchers to examine PedNet Registry data to determine if there was more of a risk of inhibitors based on blood type. The information showed that there was no significant difference in inhibitor incidence between blood group O and non-O blood groups.
FDA Approves Qfitlia (fitusiran) - a Subcutaneous Therapy for Hemophilia A or B
April 3, 2025
The US Food and Drug Administration approved Qfitlia (fitusiran) a new subcutaneous therapy that can be used for both hemophilia A and B. The therapy uses a small interfering RNA technology to target antithrombin. The therapy can be administered less frequently and more easily than more traditional treatments.
Paper Looks at Bone Health Screening Rates at HTCs Nationwide
March 27, 2025
A paper published in the journal Haemophilia showed that among HTCs that responded to a survey, only 31% recommended routine bone scans and vitamin D testing for patients. Less than half of all US HTCs responded to the survey, but responses seemed to indicate that screening for bone health is not consistent across the country.
Takeda Will Discontinue Two Treatments for Hemophilia
March 18, 2025
Takeda Pharmaceuticals announced they will discontinue two of its hemophilia treatments: HEMOFIL-M and RECOMBINATE. They shared other factor VIII products as alternative medications-contact your pharmacy if you have questions about this change.
Study: Children with Hemophilia Have Problems with Dynamic Balance and Falls
March 18, 2025
In a study published in the journal Gait & Posture, children with hemophilia were found to have more problems with dynamic balance (the ability to maintain balance while moving) and had an increased risk of falls compared to their healthy peers.
Pfizer Discontinues Hemophilia B Gene Therapy Beqvez
February 25, 2025
Pfizer has announced they are ceasing global development of Beqvez, their hemophilia B gene therapy product. The treatment was approved by the FDA in Spring of 2024.
CSL Reports on Four Years of Data for Gene Therapy Hemgenix
February 9, 2025
CSL shared four years of results from its pivotal HOPE-B study that demonstrates the long-term durability and safety of etranacogene dezaparvove-drlb (Hemgenix), the first FDA approved gene therapy for hemophilia B. Findings showed 94% of patients treated with a one-time infusion of Hemgenix were able to maintain near normal factor levels.
FDA Approves New Non-Opioid Treatment for Pain
January 31, 2025
The US Food and Drug Administration has approved suzetrigine (Journavx) 50 mg oral tablets, a first-in-class non-opioid analgesic developed to treat moderate to severe acute pain in adults. It works by targeting a pain-signaling pathway involving sodium channels in the peripheral nervous system, before pain signals reach the brain.
FDA Approves First Subcutaneous Prophylaxis for Hemophilia With Inhibitors
January 29, 2025
The FDA has approved Concizumab-mtci, known as Alhemo, the first subcutaneous therapy for the prophylactic treatment of both hemophilia A or B in adults and children ages 12 and older. The treatment offers a much needed alternative for hemophilia B patients, and adds an option for those with hemophilia A with inhibitors.
Be Biopharma Secures Financing to Advance Hemophilia B Treatment BE-101
January 17, 2025
Be Biopharma has raised $92 million in series C financing to support the first in-human Phase 1/2 trial of BE-101, the company's B-cell therapy candidate for hemophilia B. The treatment is designed to use a patient's own immune B-cells, genetically modify them, and infuse them back into the patient.
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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Previous articles featured on our website can be found at our archive page here. Please note that links in our archive files may take readers to websites that may have changed their link. In some cases these links do not work anymore. We periodically check and remove articles that have broken links. If you have questions about these articles, please contact us at hemophilia@ucdenver.edu.