When my boys were little, I dreamed about what their chosen activities might be — band, swimming, theater, or maybe soccer. I wanted to be a soccer mom, cheering on my sons from the sidelines and bringing orange slices for the team. My oldest son, Julian, did play soccer…

Sangamo Therapeutics has dosed the first patient in a Phase 1/2 clinical trial testing its investigational genome-editing therapy, SB-FIX, for patients with severe hemophilia B. The Georgetown University and MedStar Georgetown University Hospital are conducting the study, which is still recruiting participants in nine sites across the United…

A small survey study in Germany found that many people with severe hemophilia are not interested in receiving gene therapy, despite having “only general knowledge” of the treatment option — which, the researchers noted, could be “a potential cure with a single infusion.” According to the scientists, these…

Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.

Treatment with the experimental gene therapy fidanacogene elaparvovec led to significant reductions in bleeding rates for men with moderately severe to severe hemophilia B who participated in a Phase 3 clinical trial. That’s according to top-line data from the study BENEGENE-2 (NCT03861273), recently announced by the therapy’s developer,…

In this video from the Centers for Disease Control and Prevention, learn how children with hemophilia can play and live safely. The simple animation features four boys who all have hemophilia but love to play sports. MORE: How hemophilia is inherited The film advises that…

https://www.youtube.com/watch?v=qKRqJWVQRio This video from the Centers for Disease Control and Prevention takes a look at hemophilia and explains what the blood disorder means for those who suffer from it. MORE: Three tips for coping with a hemophilia diagnosis Hemophilia patients and caregivers share how hemophilia affects their everyday lives…

Mild to moderate hemophilia B affects the relationships of adult patients, children with the disease and their caregivers in aspects such as telling others about the diagnosis, selecting a partner, and feeling bullied, according to an American study. The research, “Mild-severe hemophilia B impacts relationships of…

Sometimes I forget I have hemophilia. I know that sounds silly, particularly after years of struggle to obtain a diagnosis, yet it’s true. This forgetting is great when I’m healthy and have no issues. There are times, however, when it’s imperative that I remember I have hemophilia. Sometimes I…

Researchers have developed a biodegradable capsule to deliver a protein missing in patients with hemophilia B, according to a new study published in the International Journal of Pharmaceutics. If it proves to be successful, this capsule may provide a cheaper and less painful option to injections or infusions. The…