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…

Kendall Fitzpatrick and I have much in common. Like me, she has hemophilia B and von Willebrand disease. She’s a registered nurse, works for a specialty pharmacy, and is an outspoken advocate for women with bleeding disorders. She lives in New Kent, Virginia, and is mother to a…

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…

My youngest son, Caeleb MacDonald, is a 16-year-old with hemophilia and chronic joint pain. Several times this year, the pain has become so intense that he cannot get out of bed. I cannot help but associate his new pain issues with his earlier years, when he suffered multiple…

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…

My husband, Jared, and I are in an interesting stage of our parenting journey. Our daughter, who is almost 4, will be going to preschool soon, but for now, she spends almost every day with us at home. She has a rather introverted temperament, so most of the…

Two new gene therapy vectors designed for liver diseases show promising effects in a mouse model of hemophilia B, according to a recent study. The study, “Adeno-associated virus serotype 2 capsid variants for improved liver-directed gene therapy,” was published in Hepatology. Hemophilia B is caused…

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…