Dosing has begun in a second group of hemophilia B patients enrolled in a Phase 1/2 dose-confirmation trial of the experimental gene therapy FLT180a, the treatment’s developer, Freeline, announced. The dose determined to be optimal in this study is expected to finalize plans for a pivotal Phase 3…

When I was younger, I often yearned for a fast-paced life. As a teenager, I rarely got to leave the house by myself. I coped by working on novels while locked away in my hot, stuffy bedroom. I loved writing in silence, but a greater part of me really wanted…

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…

It finally happened. COVID-19 found me. After two and a half years of following the guidelines, I couldn’t outwit the virus. I had two vaccines and a booster on time, and I declined invitations. I wore a mask in Publix and Walgreens even when nobody else was doing it.

As summer begins, the MacDonald family continues a great tradition of visiting a local museum every Friday. We started at least 12 years ago when my oldest son, Julian, graduated middle school. Caeleb, my youngest boy, continues our special ritual and looks forward to exploring the many things that…

My husband and I are both United Methodist clergy, so our children are called PKs (pastors’ kids). Some PKs earn reputations for misbehaving, giving their parents plenty of gray hairs. Other PKs are kind and considerate, attend youth groups and Bible studies, and are seen as model children. My sons…

Children and young adults with hemophilia A and hemophilia B have several behavioral problems, ranging from depression and anxiety to aggressive behaviors, a small study suggests. Age, disease severity, and joint disease duration were found to be significantly correlated with several of these problems. The findings were reported in the…

My husband, Jared, and I work to earn a living. We freelance for various clients and run a business — though due to the state of the economy here in the Philippines, business isn’t as profitable as it used to be. In between jobs, we dedicate a significant chunk of…

Preventive treatment with a low dose of factor replacement therapy in children with hemophilia A led to sufficient levels of the clotting factor in most patients, but the time in which it did so varied, a recent study reported. Researchers noted that while findings support the use of this…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa, an experimental replacement therapy for hemophilia A designed for once-weekly dosing. This designation is given by the FDA to help speed the development and review of treatments for serious or life-threatening conditions. Specifically,…