Gene therapy has long been touted as a revolutionary hope for people with hemophilia B, promising a one-time treatment to drastically reduce — or even eliminate — the need for frequent infusions. Yet that hope has hit another roadblock. Pfizer announced last week that it’s halting development of…
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Catalyst Biosciences has received a European Union patent for its investigational hemophilia B treatments, including its lead therapy candidate, dalcinonacog alfa (DalcA) and a preclinical gene therapy candidate called CB…
Gene therapy delivered to muscle was able to completely correct hemophilia B deficient blood clotting in a safe and well-tolerated manner for several years — in animal models of the condition. The study with finding, “Complete correction of hemophilia B phenotype by FIX-Padua…
Poor joint health is associated with a higher risk of low self-esteem in adolescents with hemophilia, a study has found. The study, “Assessment of Self-Image With the Offer Self-Image Questionnaire in Adolescents With Hemophilia: A Single-Center Experience,” was published in the Journal of Pediatric Hematology/Oncology.
Raising two sons with severe hemophilia A has been a journey filled with ups and downs, joys and fears. I lived in constant fear when my first son, Julian, was a baby. Not only was I concerned about feedings, diaper changes, and making sure he was breathing in…
When you have a year that is best left in the past, ushering in the new year is a time of celebration. You start with a clean slate and an attitude of hopefulness for what is yet to come. For many years I based the quality of a…
Allyx Zurio Formalejo and her husband, Jared, open up about life with hemophilia B and parenting their daughter, Citi. They share how independence, humor, and everyday moments — like helping with transfusions — shape their family’s perspective on disability, resilience, and joy. Read more in HemoWife.
The Fitbit wristband can help with the continuous monitoring of physical activity in people with hemophilia, but other strategies are needed to encourage increased activity, a study suggests. The study, “Physical Activity Monitoring and Acceptance of a Commercial Activity Tracker in Adult Patients with Haemophilia…
Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found. The study, “Patients’ and parents’…
The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s investigational one-time gene therapy BBM-H901. According to the company, BBM-H901 is the first investigational gene therapy given intravenously, or directly into a vein, for hemophilia B or any genetic disease…