Regular and sustained physical exercise — including aerobic activities, strength training, and balance exercises — may help ease chronic pain, improve physical performance, and enhance quality of life in people with hemophilia of all ages, according to a review study. The researchers found that exercise is generally safe when…
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World Hemophilia Day, an annual effort to raise awareness of hemophilia and other bleeding disorders, is on April 17, and this year, the focus is on the importance of a prompt, accurate diagnosis as a key step toward achieving good health for people…
Preventive treatment with Hemlibra (emicizumab) is associated with better bleeding control and lower medication costs compared with immune tolerance induction, or ITI, among children with severe hemophilia A with inhibitors, a new study in China found. The researchers noted that use of the injection therapy Hemlibra also led…
A single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox) continues to prevent bleeds and the need for preventive treatment (prophylaxis) for up to five years in most treated men with severe hemophilia A, according to final Phase 3 trial results. Activity of factor VIII (FVIII), the clotting…
Measuring levels of YKL-40 — a protein associated with inflammation and tissue remodeling — may help detect hemophilic arthropathy, the joint damage caused by repeated bleeding, in people with severe hemophilia A, a study found. The researchers said the results “suggest that YKL-40 has potential as a biomarker, with its…
People with hemophilia who report better mental health may be more likely to follow their prophylactic (preventive) treatment as prescribed, according to a new study based on a survey of 78 adolescent and adult males in Brazil. Researchers said this finding “underscores the need for [clinicians treating people with…
Grand Life Sciences Group has entered into an exclusive collaboration agreement with Belief Biomed (BBM) to commercialize BBM-H803, BBM’s one-time investigational gene therapy for hemophilia A, in Greater China if it is eventually approved in the region. The agreement covers Mainland China, as well as Hong Kong,…
The one-time gene therapy BBM-H901 (dalnacogene ponparvovec) has been officially approved in the Macao region of China for the treatment of adults with moderate to severe hemophilia B, thus expanding access to the medication beyond mainland China. Developed by Belief Biomed (BBM), BBM-H901 became China’s first and only…
A small survey study in Germany found that many people with severe hemophilia are not interested in receiving gene therapy, despite having “only general knowledge” of the treatment option — which, the researchers noted, could be “a potential cure with a single infusion.” According to the scientists, these…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
