Vision Outcomes in Children Treated With Voretigene Neparvovec for -Associated Retinopathy

What are the vision outcomes in young children with -associated retinopathy after voretigene neparvovec (VN) treatment?In this case series of 14 young children, VN gene therapy showed improvement of pattern visual evoked potentials (VEPs) in the majority of children, worsening in only in patients who showed localized chorioretinal atrophy.Results suggest that pattern VEPs objectively indicate functional recovery of the retinogeniculostriate pathway after VN treatment in young children with -associated retinopathy; these findings support the use of pattern VEPs as an outcome assessment in future pediatric retinal gene therapy trials, enhancing intertrial treatment evaluation and accelerating translation into practice.

Corresponding Author: Dorothy A. Thompson, PhD, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London WC1N 3JH, United Kingdom (dorothy.thompson@ucl.ac.uk).

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