New evidence suggests that a parameter called FVIII-EC50 has potential as a new way to predict the response of an individual with severe hemophilia to preventive treatment with FVIII, according to a small study. The study “Evaluation of EC50 of factor VIII as predictor of prophylaxis efficacy in patients…

Valoctocogene roxaparvovec, BioMarin Pharmaceutical‘s investigational gene therapy, results in a more than 90% drop in annual bleeding rates and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, updated data from a Phase 1/2 clinical trial show. It also precluded the need for…

What do developmental disabilities, French bread, endometriosis, nutrition, English tourism, and bleeding disorders have in common? They all claim March — or a select day or week during the month — for awareness. Organizations dedicate this time to educating the public through local and national gatherings and social media campaigns.

“It’s been too long.” “Oh my goodness, your kids have grown!” “It’s been forever since we’ve seen each other.” These sentiments resonated as members of Sangre de Oro, the Bleeding Disorder Foundation of New Mexico, gathered for Family Education Weekend March 21-23 in Albuquerque. While many chapters around the…

Note: This story was updated July 20, 2020, to note that Hemlibra binds to activated FIX and FX, rather than to FIX and FX. When given as preventive treatment to avoid spontaneous bleeds in people with hemophilia A with inhibitors, Hemlibra (emicizumab) continues to show a…

An investigational gene therapy for hemophilia B, called CB 2679d-GT, increased factor IX (FIX) levels and significantly reduced bleeding in a mouse model of the disease, a preclinical study demonstrated. The clotting activity of this potential therapy, being developed by…

When a spouse or partner has a bleeding disorder like hemophilia, occasionally there may be situations that make us wonder if they are at fault for an injury they’re battling. Perhaps they went beyond their maximum physical capacity in sports or at work. Maybe they got caught up…

Note: This column describes the author’s own experiences with Mounjaro (tirzepatide). Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. In a previous column, I shared my journey of embarking on a weight loss program using the type…

Looking at myself now, my younger self never would have expected me to be where I am. Recalling my younger years, I remember having anxiety about being alone when I grew up. I always thought I’d have a hard time finding a partner and that my dating life would be…

My husband, Jared, lives with severe hemophilia B and a seizure disorder. Despite these challenges, he’s active in ways that many people wouldn’t expect. He swims, lifts weights, and plays badminton. He can also keep up with our 5-year-old daughter, who is a huge ball of energy. We recently…