European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…

I’m grieving for my youngest son, Caeleb, a high school senior. Caeleb’s academic pursuits haven’t been successful, according to the world. Society revels in straight A’s, being in the top 10% of a graduating class, and receiving full-ride scholarships to college. If a student isn’t meeting these expectations and doesn’t…

Hemophilia is a rare inherited disease where a person is lacking a certain type of clotting factor. This means that their blood is unable to clot so injuries will result in extensive bleeding. If left untreated, this bleeding can become life-threatening. Here are a few fast facts about this rare…

I was at the Coalition for Hemophilia B’s Annual Symposium this month in Orlando, Florida — surrounded by my community, sharing meals and meaningful conversations — when it happened. No bump. No injury. No accident. I was literally just standing in the elevator when I felt it: a sudden,…

Officials of the U.S. Food and Drug Administration and Alnylam Pharmaceuticals have agreed on safety measures and a “risk mitigation strategy” to allow the resumption of clinical studies of fitusiran, the company said in a recent press release. All ongoing trials of fitusiran had been suspended after the Sept. 7 death…

After five years of follow-up, Hemgenix (etranacogene dezaparvovec-drlb) was associated with durable factor IX (FIX) levels, lower bleeding rates, and a substantial reduction in the need for regular FIX infusions, with a generally favorable long-term safety profile in men with severe or moderately severe hemophilia B. These data…

A first patient has been dosed in a Phase 1/2a clinical trial that’s assessing the safety and preliminary efficacy of ASC618, a second-generation gene therapy for hemophilia A. The Phase1/2a study (NCT04676048), which has been in the works since 2021, aims to enroll about 12 men with severe…

Treatment with platelets stripped of their natural sugar molecule coating helped to prevent inhibitors, or neutralizing antibodies, from forming against clotting factor VIII (FVIII) replacement therapy in a mouse model of hemophilia A, a study reported. Platelets promote blood clotting, and they are generally thought to boost inflammation…

Facial and scalp swelling can be indicators of childhood hemophilia, but for children who are misdiagnosed and treated incorrectly, the effects of the disease’s elevated bleeding predisposition can be catastrophic. In the study, “Facial and Scalp Swelling in the Pediatric Population With Hemophilia: A Diagnosis Pitfall,” published in…