Patricia Inácio, PhD,  science writer—

Parents of children with hemophilia in the U.K. are generally aware of gene therapy, but many lack both the knowledge necessary for an informed decision and confidence regarding this form of treatment, according to a questionnaire and interview-based study. Most of those interviewed were unaware that gene therapies are…

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…

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…

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,”…

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…

Long-term treatment with Esperoct (turoctocog alfa pegol) prevented bleeding in one-fifth of children with severe hemophilia A over the course of a Phase 3 clinical trial, according to final results from the study. Treatment was effective in both spontaneous and traumatic bleeds, and appeared to be…

An upcoming episode of the TV series “Advancements,” hosted by Ted Danson, will be dedicated to current and future research and therapeutics for inheritable bleeding disorders. The new episode will feature work developed by the National Hemophilia Foundation (NHF) and explore the non-profit’s programs in bleeding diseases, such…

The National Hemophilia Foundation (NHF) has opened a grant program to support the local groups and chapters that work with bleeding disorder families, and have watched as their revenue sources dry up during the COVID-19 pandemic. Called the COVID-19 Fund Bridge Grant program, the effort is open to…

Starting factor VIII (FVIII) preventive therapy before the age of 2.5 years improves joint outcomes in young adults with hemophilia A compared to starting treatment later in childhood, a clinical trial shows. The study, “ Young adult outcomes of childhood prophylaxis for severe hemophilia A:…

Under-the-skin, preventive treatment with Dalcinonacog alfa (DalcA) led to sustained and protective levels of factor IX (FIX) in people with severe hemophilia B, final data from a Phase 2b trial show. The findings, Phase 2b Trial to Evaluate the Safety and Factor IX Levels of a Daily Subcutaneous Prophylaxis…