Jivi (BAY94-9027), a lab-made version of the blood clotting factor VIII (FVIII), has been approved by the European Commission for the prophylactic (preventative) treatment of hemophilia A in patients older than 12, Bayer announced. The recommended prophylaxis for Jivi administration is every five days, every…

Gene therapy has the potential to stop hemophilia in its tracks. But such therapy will likely be costly, and can carry risks that have generated some apprehension among patients who could benefit most from it. That’s the message from hemophilia patient advocates who spoke at the 2018 NORD Rare Diseases…

A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…

The workshop and production process over, it was curtains up for “Hemophilia: The Musical,” a unique theatrical event starring high school students affected by bleeding disorders. Performed at New World Stages in New York City on Nov. 12, the premier Broadway-style production rounded out a three-day theater and arts…

An important part of my role in the Hemophilia Association of the Philippines for Love and Service (HAPLOS) is the promotion of physical fitness and exercise. I see many young hemophiliacs strapped to wheelchairs and wearing protective gear. It pains me to see them deprived of the ability…

At 28 weeks pregnant, it seems I have entered my “nesting” phase. Two days ago, my husband Jared and I chanced upon a good deal for a crib. We had no intentions of getting a crib then, but by a stroke of serendipity, we ended up with one anyway.

uniQure is continuing to enroll participants for its global Phase 3 trial (NCT03569891) testing the potential gene therapy AMT-061 in men with severe or moderately severe hemophilia B, according to an update from the company. In addition, uniQure announced that three patients included in a Phase 2b trial (…

Bioverativ will present some of its most recent therapeutic advancements regarding hemophilia and other blood diseases at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego, California, Dec. 1-4. The company, which is part of Sanofi, has focused on the development and commercialization of innovative…

Aptevo Therapeutics provided an update on Ixinity, its hemophilia B therapy, that includes developing a larger 3,000 IU vial assay for patient convenience. The company also plans to initiate a Phase 4 clinical trial in a pediatric patients, and to apply for Ixinity’s approval outside of the United States.

“Write about the first time you witnessed me with a bleed,” offered my husband Jared as we talked about my column over breakfast. Couples usually remember firsts. The first date. The first kiss. The first of anything special or unique to both of them, including the good and…