As my 30th birthday approached, I found myself grappling with a whirlwind of emotions. Unlike my husband, Jared, who greeted his own 30th with gratitude for another year of life, I was consumed by crippling anxiety. The looming pressures of being a mother and caregiver — Jared has…

My 24-year-old son, Julian, lives out of state, where he attends college and works as a barista. He pays his bills and “adults” well, making me immensely proud of him. When he was a teenager, I didn’t think I would live to see his adult years. Like most teenagers who…

I love the smell of a freshly painted room. The roller soaks up the new color as it moves around in the pan, waiting to be rolled onto the wall in big, broad strokes. The walls must desperately need a fresh coat of paint to soak up the new color…

Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…

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…