Evaluation of early prophylactic corticosteroid administration on early safety and efficacy outcomes of AAV gene therapy in the hemophilia dog model.
INTRODUCTION Prior to commercial withdrawal, adeno-associated virus (AAV) gene therapy was approved for the treatment of adults with severe hemophilia A. Mechanisms underlying variability, durability, and liver transaminitis are largely uncharacterized. OBJECTIVES To evaluate the effects of prophylactic corticosteroi...