Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…

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…

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to rofecoxib (TRM-201), an non-opioid investigational pain treatment for hemophilic arthropathy (HA, a hemophilia degenerative joint disease) under development by Tremeau Pharmaceuticals. Rofecoxib is a COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile. It is…

I first attended a Bleeding Disorders Conference in October 1998. The National Bleeding Disorders Foundation (formerly the National Hemophilia Foundation) offered the event during Halloween week in New Orleans. My wife, Cazandra; my oldest son, Julian; and my mother, Ruby, entered our hotel with me, not sure…

When I was diagnosed with hemophilia B and von Willebrand disease in the 1970s, I was just 10 years old. Back then, most people didn’t believe girls could have hemophilia. I was told that my “deep blood” didn’t clot properly and sent on my way. Bleeding…

Treatment with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) can reduce bleeding frequency and the use of replacement therapies, while maintaining or improving the quality of life of people with severe hemophilia A for up to five years, updated Phase 1/2 trial data show.  “These…

My youngest son, Caeleb, called me to pick him up early from jazz band rehearsal last week. I could hear a hint of panic in his voice. Caeleb has complained of arm pain over the past few months, but it never seemed to be a big problem. I hoped it…

A participant in a Phase 3 clinical trial evaluating Roctavian (valoctocogene roxaparvovec), a gene therapy for hemophilia A, has been diagnosed with leukemia. A genetic assessment of the case, conducted by the therapy’s developer, BioMarin Pharmaceutical, suggested the cancer diagnosis was not linked to Roctavian. Details of…

I’m standing in line at a convenience store after my appointment at the salon. It’s almost lunchtime and my stomach is grumbling, as I left our condo without having eaten breakfast. My husband, Jared, who has hemophilia and a seizure disorder, had a series of seizures the day before…

AAV5 DetectCDx, a companion diagnostic test used to determine patient eligibility for the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox), has been cleared for use under more stringent European Union rules coming into effect in a few years. Specifically, the test gained the Conformité Européenne (CE) mark under…