Weekly and monthly treatment with Mim8, an investigational antibody therapy, was seen to be better than on-demand or standard preventive treatments at reducing bleeding episodes in patients with hemophilia A, regardless of inhibitor status, who took part in a clinical trial. Top-line results from the global Phase 3…
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Qfitlia (fitusiran) is an RNA-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with or without inhibitors. It is given via under-the-skin, or subcutaneous, injections that can be self-administered.
I recently came across a social media post that hit home. It began: “Never judge someone with a chronic illness if they …” and went on to list things like skipping meds, eating foods that might trigger symptoms, resting all day, or even doing something that looks “wrong”…
A global survey of more than 2,700 hemophilia patients, caregivers, and healthcare providers, which aimed to uncover the daily challenges of living with the rare blood disorder or caring for those who do, found that many patients regularly hide their symptoms — including from their doctors. “More than…
Jim Christensen Jr., a hemophilia A patient who has supported the National Hemophilia Foundation (NHF) for more than three decades, has received the Lester A. Rosen Humanitarian and Achievement Award, given annually by Ameritas, for which he is an independent financial representative. With the award, the insurance company…
A gene therapy being developed by ASC Therapeutics restored levels of the clotting factor missing in hemophilia A in a mouse model of the disease, and further tests in primates and human cells suggest the therapy might also work for people, the company announced. Results from…
3 Tips for Coping With a Hemophilia Diagnosis
Discovering your child has hemophilia can be devastating. While you will certainly need to make some lifestyle changes moving forward, with the correct treatment and preventative measures, there’s no reason why your child can’t live a happy and healthy life. To help you come to terms with life with hemophilia and…
A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with severe hemophilia A. These are the latest results from the five men given the highest dose of the…
SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual bleeds, according to preliminary data from an ongoing Phase 1/2 trial. The trial (NCT03003533) is intended to explore the safety and efficacy of a single intravenous…
Garrett Hayes, who is studying medicine in Texas and has a severe form of hemophilia A, is the first recipient of the National Hemophilia Foundation’s (NHF) Jason Fulton Memorial Scholarship. The scholarship honors the memory of Jason Fulton, a hemophilia patient who died in 1995 at age…