Years ago, a home health nurse came to my house to infuse my medication for von Willebrand disease and hemophilia B. Treatment for my bleeding disorders is often administered intravenously, meaning it’s injected directly into a vein. I’d had surgery a few days before and needed the…

Proteins from tardigrades — microscopic animals known for their ability to survive in extreme conditions — could be used to stabilize replacement therapies for hemophilia A, allowing treatments to be stored for longer periods of time without refrigeration, a proof-of-concept study shows. “Our work provides a proof of principle…

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…

Hemophilia A patients at a single center in France reported high rates of satisfaction with Hemlibra (emicizumab) after a year of real-world use, a study has found. Patients reported improvements in health and life quality, as well as reductions in pain and bleeding with the therapy, but problems…

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…

Most people with severe hemophilia A who were given once-weekly treatment with efanesoctocog alfa in the Phase 3 XTEND-1 trial were bleed-free over a year on treatment. The therapy also improved joint and overall physical health and eased pain intensity in the participants compared with previous treatments. Results…

Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe hemophilia A in the Phase 3 GENEr8-1 study were off standard replacement therapies and did not experience any bleeds requiring treatment. That’s according to new data announced in a…

The U.S. Food and Drug Administration (FDA) has cleared CRYOcheck Chromogenic Factor IX assay, a test intended to be used by clinical labs to detect a deficiency in factor IX (FIX), the cause of hemophilia B. The chromogenic, or color-based, test developed by Precision BioLogic is reported…

The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical would produce the gene therapy Roctavian (valoctocogene roxaparvovec) if it’s approved. The agency is reviewing a biologics license application (BLA) from BioMarin that seeks approval of Roctavian to…

Patients with moderate to severe hemophilia A who switched to Kovaltry (octocog alfa) showed no worsening of their annual bleeding rate, according to real-world data from a Phase 4 study. Adhering to the therapy remained high and patients reported high treatment satisfaction. Overall, the study “confirms and extends clinical trial…