A European Medicines Agency (EMA) committee has recommended the approval of Novo Nordisk’s concizumab as a prophylactic, or preventive, treatment for adults and children, ages 12 and older, with hemophilia A or B who have inhibitors. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use…

I live in the high desert of New Mexico, where we have over 300 days of sunshine each year. It’s a perfect place to hike, bike, climb, and run. The beauty of this place is undeniable. Yet despite all its splendor, I often find myself yearning for the storms.

When you’re a parent to a child newly diagnosed with a bleeding disorder, the immediate concern is for your child. What do I do next? Will they be OK? What will life look like from now on? You find yourself searching for answers and developing a support system…

My biggest fear for my sons, who live with severe hemophilia, is an auto accident. When my oldest son, Julian, began to drive, I impressed upon him anew the importance of wearing a MedicAlert emblem. He wore it like dog tags for years when he was younger,…

BioMarin Pharmaceutical Inc. and Believe Limited have announced the students chosen to participate in the theatrical production of “Hemophilia: The Musical,” a program that aims to empower the bleeding community through performance while raising public awareness of the connection between arts and wellness. The 25 high school students hail from…

Hemophilia is a blood disorder that’s usually genetically inherited from parents. People born with hemophilia have little or no clotting factor – a protein needed for normal blood clotting. Hemophilia can also be acquired, meaning patients don’t inherit the disease from their parents but instead develop it at some point during…

Some of my most enjoyable moments are guy times with my boys. We laugh, we attempt to keep the house in order, and we succeed, despite ourselves. My sons, who have my blood running through their veins, remind me that life will be OK; that any problem can be solved,…

Hemlibra (emicizumab-KXWH) safely reduces bleeding episodes in people with hemophilia A without inhibitors, resulting in a high rate of patient satisfaction, according to a review of published studies. “The success of [Hemlibra] underscores the potential for innovative therapeutic approaches to significantly improve patient outcomes and quality of life…

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, “…

AMT-061 (etranacogene dezaparvovec), uniQure‘s investigational gene therapy for hemophilia B, is safe and effective — even in patients with pre-existing antibodies against the treatment’s viral carrier, according to new trial data. Data from the Phase 3 HOPE-B trial suggest that nearly all hemophilia B patients, regardless of pre-existing viral…