Despite the increasing use of preventive factor VIII (FVIII) replacement therapy, people living with hemophilia A in Japan still experience a significant disease burden, according to a recent study. Using information from health records databases, researchers found that while patients have used increasing doses and spent more on medical…

I like my cabinets neatly arranged. But the kitchen cabinets are often hit-or-miss in the organization department. For example, the cabinets that house plastic lids and containers can be treacherous. When organized, the cabinet allows us to see what’s available. Every cover matches its proper container, and items sit neatly…

Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then move in pivotal clinical trials of SerpinPC — an investigational therapy for hemophilia A and B patients, regardless of disease severity or inhibitor status — that it plans to launch…

Enzyre has raised €12 million (over $12 million) to speed the development of EnzyPad, a device that could one day allow people with hemophilia A to monitor their blood-clotting status in real time from anywhere, including their home. “We are delighted to have closed this successful financing…

There’s a freedom children have when they swing. Sometimes they need a little push, but before long, they learn how to push themselves. The sounds of chains squeaking and shoes scraping the dirt are imprinted in my mind. Swinging higher and higher is always the goal. Hair flies in the…

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…

I recently watched the eight-episode Canadian drama miniseries “Unspeakable,” which was inspired by real events and first televised a few years ago. The series is the creation and passion of Robert C. Cooper, a writer and producer best known for his work on the television series “Stargate…

Apologies aren’t easy. Sometimes when hurtful words are shared, an apology is in order. Likewise, a mistake that affects another person is also a time when an apology is needed. But why do people apologize when something is out of their control? My youngest son, Caeleb, is 16 and lives…

From the time my wife found out she was pregnant, we celebrated a ritual each evening until the birth of my oldest son, Julian. Before we kissed good night, I put my face to her belly and sang the song “For Baby (For Bobbie)” to him. Without seeing…

The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…