Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…
A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…
A gene therapy-based factor VIIa (FVIIa) given as prophylactic, or preventative, treatment was seen to produce specific factor levels needed to reduce the frequency of spontaneous bleeding episodes in a rat model of hemophilia A. According to scientists, this work may be a first step in developing therapies that…
Doctors in Canada are proposing a set of measures to better judge outcomes and the success of programs helping teen-age hemophilia patients transition to adult care, especially for those with more severe disease. They also listed 15 “key elements” of a successful pediatric-to-adult transition program, including eight the doctors saw as feasible…
Recognizing risk factors of thrombosis and carefully monitoring medication doses are crucial for minimizing thrombotic complications in older patients with acquired hemophilia and serious illnesses like cardiovascular disease, a report describing four cases says. The study, “Acquired Haemophilia A. Which is the best therapeutic choice in older adults? Single…
The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…
A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…
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…
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,”…
People with hemophilia may benefit from physiotherapy and rehabilitation programs intended to manage bleeding and joint disease (arthropathy), but more research is needed to better understand those programs — and their duration — that might be of most help, a review study says. The review, “Recent advances in musculoskeletal…
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