News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to provide…

Catalyst Biosciences has announced new clinical data supporting the safety and efficacy of its lead candidate therapies for treating hemophilia. Factor IX (FIX) dalcinonacog alfa (DalcA) was found to safely prevent bleedings in adults with hemophilia B, while factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) was…

Joint changes detected by magnetic resonance imaging (MRI), and often are missed on radiographs, are a strong predictor of damaging bleeds over the next five years in joints of people with hemophilia, a study suggests. A 10-fold increase in the five-year risk of joint bleeds was found by…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Researchers have developed a faster, less expensive, reliable method for non-invasive prenatal diagnosis (NIPD) of hemophilia A. Future, larger studies are required to confirm the utility of the method, which could be used for the NIPD of hemophilia A and type B, as well as other X-linked…

Aptevo Therapeutics announced the dosing of a first patient in a Phase 4 study aiming to extend use of its prophylactic (preventive) treatment Ixinity (coagulation factor IX [recombinant]) to children with hemophilia B  younger than 12 years old. “Initiation of the clinical study of Ixinity in…

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.