A single infusion of AMT-061 (etranacogene dezaparvovec), uniQure’s experimental gene therapy for the treatment of hemophilia B, led to increased and sustained activity of clotting factor IX (FIX), according to one-year data from a Phase 2b clinical trial. The therapy effectively prevented…
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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…
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…
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.
In recognition of World Hemophilia Day (WHD), Catalyst Biosciences is hosting an “Ask Me Anything” (AMA) session on Reddit on April 17. During the Reddit AMA event, which will take place at noon EST Wednesday, clinicians will answer questions from patients and caregivers about bleeding disorders including hemophilia. A…
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…
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…
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…
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…
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.