Author Archives: Steve Bryson PhD

Risk of Inhibitor Development Tied to High BAFF Protein Levels

Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…

Enrollment Complete for GENEr8-3 Trial of Roctavian Plus Steroids

BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…

Preventive Esperoct Plus Surgery Reduces Joint Bleeds, Aids Mobility

Preventive treatment with Esperoct (turoctocog alfa pegol) plus joint surgery significantly decreased annual joint bleeding rates, reduced pain, and improved mobility in hemophilia A patients, according to an analysis of Pathfinder trial data. “Our results therefore support the notion that patients with haemophilia A being treated with [Esperoct]…