News

A publicly available, patient-reported outcome measure is being jointly developed by Green Park Collaborative (GPC) and the National Hemophilia Foundation (NHF) to evaluate the effect of gene therapy on the mental health outlook of people with hemophilia type A and type B. The collaboration follows work…

Specific genetic changes may account for a larger proportion than previously thought of children with severe hemophilia B who develop inhibitors against coagulation factor IX replacement therapy, according to an international study. The study, “Inhibitor Incidence In An Unselected Cohort Of Previously Untreated Patients With…

The likelihood that teenagers and young adults with hemophilia will use a prophylactic replacement therapy as prescribed is mostly influenced by their level of responsibility in taking their infusions, and their own estimates of risk for each activity, a study suggests. To improve treatment adherence, doctors should discuss its importance during…

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.

Prophylactic, or preventive, use of Hemlibra (emicizumab) promotes joint health in people with hemophilia A, according to a new analysis of data from a Phase 3 trial. These findings were announced in the oral presentation “Bone and Joint Health Markers in Persons with Hemophilia A (PwHA)…

BioMarin is considering pricing its hemophilia A gene therapy Valrox (valoctocogene roxaparvovec), should it be approved, at $2 million to $3 million. That range would make Valrox the world’s most expensive one-time therapy. But, the company argues, this first gene therapy for any form of inherited hemophilia could…

Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.

ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good Manufacturing…

Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead programs hemophilia A and phenylketonuria into investigational new drug-enabling clinical trials. Gene therapy is an investigational treatment technique that uses genes or genetic material to treat or prevent…

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…