Today my hematologist complimented me. She told me that, had I not persevered, I may have only been labeled a “carrier” and my bleeding disorder may not have been adequately understood. For me to be healthy, it is critical that my bleeding disorder is understood. My hemophilia…

A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene roxaparvovec to this first patient seropositive for the AAV5 capsid is an important next step in our plan to…

Catalyst Biosciences has begun enrolling patients in a Phase 2/3 clinical trial of marzeptacog alfa (activated) as a preventive treatment for hemophilia A or B. Some patients develop immune responses — or inhibitors — to clotting factor treatments, preventing the therapies from being able to stop bleeding. Catalyst’s therapy, also known…

Ixinity (trenonacog alfa) has been approved by the U.S. Food and Drug Administration (FDA) for use in children younger than 12 with hemophilia B. This label expansion by the FDA means that the factor IX (FIX) replacement therapy, sold by Medexus Pharmaceuticals, can now be used in…

Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…

Some of my most enjoyable moments are guy times with my boys. We laugh, we attempt to keep the house in order, and we succeed, despite ourselves. My sons, who have my blood running through their veins, remind me that life will be OK; that any problem can be solved,…

This month, I attended One Drop, a “rare bleeding disorders consortium” hosted by Comprehensive Health Education Services (CHES) in Tampa, Florida. The event supported individuals and families with deficiencies of factor I, II, V, VII, X, XI, XIII as well as rare platelet disorders, such as Glanzmann thrombasthenia. Janet Brewer,…

Scientists in Taiwan have created a new point-of-care device to diagnose hemophilia type A and determine a person’s blood type using just a small sample of blood. The device “is especially suitable for usage in emergency or natural disasters to provide quantitative testing in rescue and relief operations,” the…

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…