Saturday afternoon marked the opening of the virtual edition of the National Hemophilia Foundation’s Bleeding Disorders Conference. I immediately missed the in-person experience, meeting old friends, walking through the exhibits, and other benefits that come with traveling to another city. But I like the opportunity to hear great thinkers in…
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Using Hemlibra (emicizumab) as prophylaxis lowers treatment costs considerably for people with hemophilia A compared to preventative factor (FVIII) treatment, according to an economic model study. The model predicts $47,814 in savings to U.S. payers in year one, and a potential for more $3 billion in savings over 20 years…
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…
People with severe hemophilia A who have two or more self-described problem joints — a person-centric characterization of joints based on underlying joint damage, pain, and mobility impairment, regardless of bleeding — have worse clinical outcomes, including more chronic pain, a new study from researchers in Europe has found.
Although gene therapy for hemophilia has reached clinical testing in humans, researchers continue to work toward improved delivery systems of the treatment — not an easy task considering the large size of the human factor VIII gene. In a study titled “Characterization of Adeno-Associated Viral Vector-Mediated Human Factor…
As a hospice chaplain, I meet people during the hardest moments of their lives. Whether at a deathbed or in a funeral home preparing for a service, these are times of finality. Grief brings people together as they share their loss and honor the person who has died. A recent…
The Arizona Hemophilia Association (AHA) is inviting camp professionals in the bleeding disorders and hemophilia communities to participate in the National Camp Exchange (NCE) program. Applications are open until May 30 for 2018 summer programs. NCE is set up to give those who plan, organize, and operate…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…
A daily infusion of low-dose factor VIII (FVIII) improves trough levels in patients with severe hemophilia A — compared to infusions performed every other day — without increasing factor VIII consumption, or the amount of medication a patient must take, researchers have found. Trough levels are the lowest concentrations of a…
Researchers at South Dakota State University (SDSU) studying mouse sperm found that drugs such as those used to treat HIV infection suppress L1s, or snippets of DNA that cause gene mutations. L1s are called transposons or mobile genetic elements. They are DNA fragments in the genome that replicate themselves and…