Last week, my husband, Jared, experienced a bleed above his knee joint. We were out on a day trip when he noticed the first symptoms. He felt a tightness in his knee, which he initially attributed to climbing several flights of stairs. It wasn’t painful for him, but as…

MRI screening can identify subclinical joint bleeding in some patients with severe hemophilia A on preventive treatment, according to a study in the Netherlands. Subclinical bleeds occur without symptoms and usually cannot be detected by standard physical and ultrasound examinations. “These MRI findings support the hypothesis that subclinical…

I remember watching “The Wizard of Oz” as a little girl. While the visual effects now appear primitive compared with today’s high-tech productions, the movie scared me when I was a kid. The scene where Dorothy was trapped in her house as the tornado lifted it into the sky seemed…

The U.S. Food and Drug Administration (FDA) is extending its review of BioMarin Pharmaceutical’s request to approve the gene therapy Roctavian (valoctocogene roxaparvovec) for adults with severe hemophilia A. A decision had been expected by the end of this month, but that date has now been…

Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…

Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available.  Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…

The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for…

February is traditionally known as the month of love. Yet while Valentine’s Day is over, the act of appreciating loved ones knows no time or place. My husband, Jared, and I have been together for almost nine years. Our relationship is somewhat unconventional — he has hemophilia and a…

Teens and young adults with moderate and severe hemophilia A on continuous preventive treatment show levels of physical activity similar to young people without the disease, but less than half of teenagers met the weekly physical activity levels recommended by the World Health organization (WHO), a study reports. “We…

In the marketing world, we have a concept called the elevator pitch. An elevator pitch is a short, persuasive speech delivered in the time it takes to ride an elevator, hence the name. The goal of an elevator pitch is to quickly and effectively communicate a person’s ideas, product, or…