People with severe hemophilia A living in Europe can now receive treatment with Hemlibra (emicizumab-kxwh) to prevent bleeding episodes. The European Commission approved the prophylactic use of Hemlibra in patients of all ages who do not have factor VIII inhibitors, announced the treatment’s manufacturer, Roche. Hemlibra is a man-made antibody…
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact on…
More than half of adults who either have hemophilia or are family members affected by the disease support the use of preconception and prenatal genetic screens, a survey reveals. However, 90 percent of this population do not agree with ending pregnancies due to hemophilia, the results also showed. Findings…
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…
Erectile dysfunction, more commonly known as male impotence, is associated with older age in men with hemophilia, especially those who smoked long term and have high blood pressure, a study reports. The study, titled “Erectile dysfunction in patients with haemophilia,” was published in Haemophilia. Hemophilia is…
Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, long-term, Japanese study. The treatment showed better results when used as a preventive, rather than an on-demand, therapy. The findings were reported in an article titled “Inhibitor development, safety and efficacy of…
Hemophilia patients who take intermediate‐dose preventive medication may experience more of an age-related decline in sports participation, joint status, and physical functioning than those on a high dose, a population-based study suggests. The study, “Sports participation and physical activity in adult…
Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…
According to hemophilia A patients, FuseNGo, Pfizer‘s pre-filled syringe device used for both the treatment and prevention of bleeding, is easy to use and has a wide range of benefits, a study reports. These results support the potential for FuseNGO to increase patient adherence to therapy. The study,…
Scientists successfully combined the gene-editing tool CRISPR-Cas9 and a special type of virus to deliver corrected genes to restore the function of the coagulation factor IX (FIX) gene in young mice with hemophilia B. The study, “Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9,”…
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