Background & Aims Spinal muscular atrophy (SMA) is an autosomal recessive, childhood-
onset motor neuron disease. Onasemnogene abeparvovec (OA) is a gene therapy designed
to address SMA's root cause. In pivotal mouse toxicology studies, the liver was identified as
a major site of OA toxicity. Clinical data reflect elevations in serum aminotransferase
concentrations, with some reports of serious acute liver injury. Prophylactic prednisolone
mitigates these effects. Herein, we aim to provide pragmatic, supportive guidance for …