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.

Nearly all men with moderate-to-severe hemophilia B given the experimental gene therapy EtranaDez (etranacogene dezaparvovec) in the HOPE-B trial have stopped using prophylactic therapies, full study results show. Trial data also indicated that EtranaDez, formerly known as AMT-061, is effective in people with antibodies against the viral vector…

It happened after a month of celebrations. First, it was Christmas and New Year’s. Then, it was my husband Jared’s birthday. Five days later, our daughter, Cittie, turned 3. Plus, we had just moved to a new home next to some uncles who kept inviting us to relatives’ birthdays…

Dexamethasone, a steroid with immunosuppressive and anti-inflammatory properties, may temporarily increase the efficacy of one-time gene therapy when given at later time points, a study in mice suggests. Lab results showed that dexamethasone increased the levels of factor IX (FIX) — the missing clotting protein in hemophilia B —…

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…

My youngest son, Caeleb, called me to pick him up early from jazz band rehearsal last week. I could hear a hint of panic in his voice. Caeleb has complained of arm pain over the past few months, but it never seemed to be a big problem. I hoped it…

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…