The U.S. Food and Drug Administration (FDA) has approved an update to the label of Alprolix, based on long-term…
Articles by Alice Melão
Administration of once-weekly Hemlibra (emicizumab-kxwh) significantly reduced bleeding rates in patients with Hemophilia A compared to other clotting…
An ongoing Phase 1/2 clinical trial evaluating a gene therapy, SPK-9001, in people with hemophilia B is expanding its enrollment…
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to…
The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s…
Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main…
Hemophilia patients are taking heart from a number of innovative treatment approaches that have reached the clinical trials stage. Researchers…
Knowledge about the clinical benefits of treatments and their importance in the outcome of patients, as well as more stable…
U.S. hemophiliacs who use patient-reported outcome (PRO) instruments can provide reliable information on their disease burden, finds a new study…
The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of…