Catalyst Biosciences has begun enrolling patients in a Phase 2/3 clinical trial of marzeptacog alfa (activated) as a preventive treatment for hemophilia A or B. Some patients develop immune responses — or inhibitors — to clotting factor treatments, preventing the therapies from being able to stop bleeding. Catalyst’s therapy, also known…

The association of hemophilia A (AHA) and myelofibrosis, a bone marrow disorder, is uncommon and rarely described but in a recent case study, researchers at the Centre Hospitalier Metropole Savoie, in France, reported a patient with AHA secondary to hematological malignancy whose rapid and challenging diagnosis — and timely treatment — was…

Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. “We are encouraged by the data from these initial Phase 3 studies demonstrating fitusiran’s potential as a new…

Hemophilia is a rare blood disease that usually occurs in males. In fact, it’s extremely rare for women to be born with the condition because of the way it’s passed down genetically. A female would need to inherit two copies of the faulty gene — one from each parent —…

Effectiveness in controlling bleeds and the potential for less frequent use of a treatment are two priorities people with hemophilia place on a potential gene therapy, and both ranked slightly higher than safety, a survey study of patients in the U.S. reports. The study, “Patient preferences and…

Creative expression within the bleeding disorders community will be on display for the thousands expected to attend the National Hemophilia Foundation’s (NHF) Annual Bleeding Disorders Conference next month in Orlando, Florida. Backed by BioMatrix Specialty Pharmacy, the exhibit, called Blood Work 2018, will showcase interactive gallery submissions from around…

The severity of a person’s hemophilia depends on the amount of clotting factor they have in their blood. Generally speaking, the disease is considered mild, moderate or severe, although around 70 percent of people living with the disease have the severe form. MORE: Five tips for healthy living with hemophilia.

Prophylactic, or preventive, treatment with factor VIII (FVIII) replacement therapy for four years reduced annual bleeding rates by 91% for people with severe hemophilia A, a study from Taiwan reported. After switching from on-demand treatment to prophylaxis, patients also experienced a 90.8% reduction in annual joint bleed rates and reported…

Life with hemophilia can be challenging but there are ways that you can manage the disease to ensure your child leads as normal a life as possible. To help you navigate your child’s illness and help prevent bleeds, we’ve put together a list of tips. MORE: How to prepare for your child’s…