I’m sitting at my desk at work, focusing on projects I must accomplish before the day ends, when suddenly my ringtone fills the room with the song “Defying Gravity” from the Broadway musical “Wicked.” I see that my oldest son, Julian, is calling me. I smile when I…

This Father’s Day, as we celebrate the role of fathers in our lives, I can think of no one more deserving of recognition in our bleeding disorders community than Iván Girón. At Genentech’s Rare Blood Disease Summit in San Francisco last year, I was immediately drawn to Iván…

The efficacy and safety profiles of preventive treatment with Hemlibra (emicizumab-KXWH) are not affected by age or the presence of some simultaneous health conditions, that is, comorbidities, in older people with hemophilia A. The findings are the result of a post hoc analysis of pooled data from Phase…

My youngest son, Caeleb, recently graduated from high school and, at 18, is legally recognized as an adult. This milestone brings a host of new responsibilities and rights: He can vote, buy and sell property, sue or be sued, and enter into binding contracts. Despite all our preparation for…

More than half the adults with hemophilia A in the Phase 3 GENEr8-1 clinical trial remain completely free of bleeds needing treatment, four years after a single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox). Also, activity levels of factor VIII (FVIII), the faulty clotting protein in hemophilia…

In real-world settings, Hemlibra (emicizumab-KXWH) can safely control bleeds in children with hemophilia A better than previous treatment regimens, a study reports. Hemlibra’s improved efficacy was more pronounced among patients with inhibitors, or neutralizing antibodies targeting factor VIII (FVIII), which is the clotting protein missing in people with…

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…

In the age of “cancel culture,” where snap judgments and public shaming abound, the journey from stigma to compassion is often fraught with challenges. I live with attention-deficit/hyperactivity disorder and bipolar II disorder, am a wife to a person with hemophilia, and formerly co-owned a business run…

The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Altuviiio (efanesoctocog alfa), a long-lasting factor VIII (FVIII) replacement therapy for hemophilia A, to include the full results from a clinical trial, the XTEND-Kids study. As requested by Sanofi, which markets…

Artificial intelligence (AI) can comb through ultrasound scans to detect joint bleeding and inflammation occurring as a result of hemophilia, a study has found, suggesting it may help doctors make quicker and more accurate diagnoses. “AI models have the potential to aid diagnosis and enable earlier therapeutic interventions, helping…