I will never forget the evening in the mid-80s when my parents sat my brother, my sister, and me down for a critical conversation. They told us that my father had HIV. There was a very good chance he would die before my sister graduated from…
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Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…
The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) asked Bayer to clarify the risks to the hemophilia community, and the potential effects, of the recent voluntary recall of two defective lots of Kogenate FS anti-hemophilic factor. In a letter sent to Paul…
The presence of inhibitors and excessive body weight increase the sense of disease burden felt by parents of children with moderate to severe hemophilia, a study reports. The study “Burden on parents of children with hemophilia: the impact of socio- demographic and child’s medical condition” was published in…
An 87-year old woman with acquired hemophilia A (AHA) showed no adverse events or major bleeding events after starting on Hemlibra (emicizumab). The findings suggest that Hemlibra may be a better option for bleeding prevention in AHA patients than bypassing agents, the team said. The study, “…
BioMarin Preparing to Submit Marketing Authorization Applications for Valoctocogene Roxaparvovec
BioMarin Pharmaceuticals is planning to submit marketing authorization applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval of valoctocogene roxaparvovec, its investigational gene therapy for the treatment of adults with hemophilia A, before the end of the…
My 13-year-old son Caeleb, who had severe hemophilia A and an inhibitor, has not had a bleed in five years. For three of those years, he received a daily infusion with a plasma-derived product, and for the past two years, he has been on a treatment that…
Bayer is recalling two lots of product vials that have been wrongly labeled as Kogenate FS antihemophilic factor (recombinant) 2000 IU, when in reality they contain Jivi antihemophilic factor (recombinant) PEGylated-aucl 3000 IU. Both Kogenate (BAY14-2222) and Jivi (BAY 94-9027) are factor VIII replacement therapies…
Preclinical Work by Sigilon Supports Cell Therapies as Durable Way of Treating Bleeding Disorders
Cell therapies may be a feasible way of achieving a long-term correction of bleeding disorders, preclinical data shared by Sigilon Therapeutics show. A single implant of Sigilon’s engineered cells given to mice enabled durable production of therapeutic levels of blood clotting factors and normalized bleeding time. The technology uses the…
Severe, moderate, and mild: These are the degrees with which a person with hemophilia is labeled based on the percentage of clotting factor in their blood. With the word “severe,” one might automatically think it is the worst kind of hemophilia. This level means that a person has less…