Using a combination of genetic and functional approaches, scientists have identified new mutations in parts of the F8 gene that do not code for factor VIII (FVIII) in a small group of French and Canadian patients with hemophilia A. These results were described in the study, “The…
Hemlibra (emicizumab) continues to safely and effectively prevent bleedings in children and adults with hemophilia A, regardless of developing neutralizing inhibitors (antibodies) against synthetic factor VIII (FVIII), according to nearly three years of data from four Phase 3 clinical trials. These and preliminary real-world safety findings from a European…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is central to NORD’s mission and history — community…
The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies. The…
Prophylactic (preventive) treatment with Adynovate safely and effectively lowers the number of bleedings, including those in target joints, in children and adults with severe hemophilia A, according to data from two Phase 3 clinical trials. Notably, personalized Adynovate treatment targeting higher minimum blood levels of factor VIII (FVIII) —…
Acupuncture was found to be more effective for reducing chronic joint pain in hemophilia patients than transcutaneous electrical nerve stimulation, or TENS, the results from a small clinical trial show. “Any treatment that can decrease chronic pain should be considered and acupuncture may have something to offer,”…
People living with bleeding disorders such as hemophilia and von Willebrand disease report consistently high levels of satisfaction with the care they receive at U.S. hemophilia treatment centers (HTCs), according to the results of the U.S. HTC Network’s first national survey. Most respondents said they were satisfied…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
Preventive treatment with Hemlibra (emicizumab) improves health-related quality of life (HQRoL) in children with hemophilia A who develop inhibitors, or neutralizing antibodies, against engineered factor VIII (FVIII), an essential blood-clotting protein, a new analysis of a Phase 3 trial shows. Such Hemlibra therapy also reduces the burden on…
Most people with hemophilia who undergo knee replacement surgery have favorable outcomes, including less pain and improved function, a new single-center study from South Korea reports. The study, “Mid-to Long-Term Survival of Total Knee Arthroplasty in Hemophilic Arthropathy,” was published in the Journal of…
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