BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…

“It’s nothing.” That phrase is often associated with women. Many have argued that women tend to obscure their feelings to avoid hurting other people, especially their partners. As a woman and a wife, I question this generalization, mostly because my husband says the phrase more than I do.

An ultrasound-mediated, non-viral gene therapy safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and lessened bleeding in a mouse model of the disease, a study shows. The delivery of a modified, improved version of the disease-associated F8 gene…

I went to college to become a teacher. Actually, to become a middle school band director. Four years of college were great, but student teaching was how I learned to teach. Two excellent supervisory teachers helped me find my way, answered questions, and taught me useful lessons by their example.

The Sacramento Christian Camp and Conference Center lies in New Mexico’s Sacramento Mountains at an elevation of 9,500 feet. It offers fresh air and magnificent views of the surrounding mountains and valleys. I’ve been visiting the camp for over 15 years and still get caught up in the extraordinary…

Spontaneous and joint bleeds, and limits on physical activity are evident in people with severe hemophilia A even when they adhere to and remain on preventive treatment, an analysis of global, real-world data showed. These findings highlight the need for additional alternative therapies for patients with severe disease. The study,…

The U.K. charity Veganuary challenges people to eat only vegan foods in the month of January as part of an effort to promote health, protect the environment, and end animal farming. People choose to follow a vegan diet, which excludes all animal products such as eggs, dairy, and meat,…

Adults with severe hemophilia B have a persistent number of bleeds per year, as well as joint problems and mild to moderate chronic pain, revealing an unmet medical need in these patients, according to real-world data from the European CHESS II study. The study, “Clinical, humanistic, and economic…

Obizur (susoctocog alfa), a pig-derived replacement therapy, can be used to prevent bleeding in people with acquired hemophilia A who need to undergo surgery, a new case report highlights. The report, “Successful perioperative prophylaxis with susoctocog alfa in a patient with acquired haemophilia A: A…

In Chinese philosophy, the concepts of yin and yang represent two opposing forces that come together in synergy to complement each other and create a perfect balance. Their symbols are interconnected black and white halves of a circle, each with a dot of the opposite half’s color. It perfectly…