An analysis was highly critical of the Orphan Drug Act, used by the U.S. Food and Drug Administration since 1983 to stimulate research into rare diseases. The study found that the act is not “sufficiently” effective in meeting the needs of patients with less common diseases, like hemophilia, while pharmaceutical companies are finding it profitable, with…

Direct‐acting antiviral (DAA) therapy is especially effective at eliminating hepatitis C virus (HCV) in people with bleeding disorders, including hemophilia, a study has found. The study, “Elimination of hepatitis C virus infection in patients with haemophilia in Belgium: A single‐centre experience,” was published in Haemophilia.

My household is navigating a season of major change. As the family of a frequently relocating pastor, we’ve honed our adaptability, mastering the rhythm of moving with grace and resilience. This summer we’ll exhibit that adaptability again as my husband moves to a new appointment in Las Cruces, New…

To encourage more women to take part in disease research, the Patient-Centered Outcomes Research Institute (PCORI) has chosen the Hemophilia Federation of America (HFA) and its FIRST project to receive a Eugene Washington PCORI Engagement Award. The HFA’s FIRST project stands for Females in Research Sharing and…

In the final days of 2023, an adult with hemophilia A named Maxwell became the first person in the U.S. to receive treatment with the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) — approved in June of last year — outside of clinical trials. “Receiving this treatment is liberating…

The National Hemophilia Foundation (NHF) has changed its name to the National Bleeding Disorders Foundation (NBDF) to reflect its support for people with inheritable blood and bleeding disorders other than hemophilia. The new name includes a new visual identity and logo. The foundation also unveiled a new tagline…

Artificial intelligence (AI) in healthcare? You’ve got to be kidding me! What a ridiculous idea! That sounds horrifying. We don’t want computers diagnosing us; we want trained, experienced professionals who can think for themselves. As someone with hemophilia, I felt this way. Strongly. Until last week. As I’ve mentioned…

As a hospice chaplain, I visit patients and their families as they are in their last months and days of life. It is humbling. Whether they’re at home or in a nursing home, these patients have something in common: They are being cared for by others. Family caregivers are the…

For people with hemophilia B, switching to Rixubis (nonacog gamma) as a factor IX (FIX) replacement therapy was not associated with increased bleeding rates, dose changes, adverse blood clotting events, or the development of inhibitors among patients with moderate to severe forms of the disease,…

Weekly use of the experimental therapy efanesoctocog alfa maintains therapeutic levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and significantly improves physical and joint health, while reducing pain, in adolescents and adults with a severe form of the disease. These are the…