Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

Spontaneous and joint bleeds, and limits on physical activity are evident in people with severe hemophilia A even when they adhere to and remain on preventive treatment, an analysis of global, real-world data showed. These findings highlight the need for additional alternative therapies for patients with severe disease. The study,…

In boys and men with severe hemophilia A, joint damage accumulates with age, and can be prevented if replacement therapy is started at a younger age, according to a small, single-center study in China. In addition, poor adherence to preventive, or prophylactic treatment was significantly associated with greater…

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…

A hemophilia diagnosis takes a toll on the social and mental health of men, who often feel misunderstood and excluded from activities such as sports, a Canadian interview-based study reports. While blood-clotting replacement therapies have improved quality of life, they can also be costly. Study participants highlighted the need…

Low-dose immune tolerance induction (ITI) — a type of treatment that can be used to eliminate inhibitors that limit the effectiveness of replacement therapies — achieved partial success in 80% of children with severe hemophilia A and high inhibitor levels, according to a recent study. The study, “…

2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia A. Under this agreement, Novo Nordisk has the option to exclusively license 2seventy Bio’s proprietary mRNA-based megaTAL technology — customizable enzymes designed to recognize and cut specific DNA regions.

Hemophilia News Today brought you daily coverage throughout 2021 of the latest scientific findings, treatment developments, and clinical trials related to hemophilia. As a reminder of what mattered most to you during this year, here are the top 10 most-read articles of 2021, with brief descriptions of what made…

Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. “We are encouraged by the data from these initial Phase 3 studies demonstrating fitusiran’s potential as a new…

I cut my thumb above my knuckle a few weeks ago. It didn’t seem like a big deal, but five Band-Aids later, I began to wonder. A few hours later, I noticed my knuckle was swollen, painful, and a bit blue. My thumb wouldn’t bend, and the swelling extended down…