S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)

Abstract

Introduction and objectivesSMA1 is a rapidly progressing neurologic disease caused by biallelic loss/mutation of the survival motor neuron 1 gene (SMN1). Onasemnogene abeparvovec (formerly AVXS-101) is a one-time GRT designed...