Patricia Inácio, PhD, science writer —

Joint pain significantly rose and overall joint health — particularly range of motion — diminished with limitations on physical activities during the COVID-19 lockdown for people with hemophilia and joint disease, a small study from Spain reported. The study “Musculoskeletal Changes in Hemophilia Patients Subsequent to COVID−19…

Intensive treatment with factor VIII, the clotting protein missing or defective in patients with hemophilia A, is associated with a greater risk of inhibitor development among patients with non-severe disease, a study has found. The findings highlight the importance of closely monitoring these patients in order to prevent or…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases. These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States:…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

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,”…