Vision Outcomes in Children Treated With Voretigene Neparvovec for <i>RPE65</i> -Associated Retinopathy
This case series study reports VN-treated patients as young as 15 months, confirming visual improvements, although not necessarily VA when off-chart VA conversions were excluded, consistent with previously reported outcomes. Pattern VEPs provided an objective measure of retinogeniculostriate recovery, supporting their use as an outcome measure in future trials for young children with inherited retinal diseases.
Authors
- Oliver R. Marmoy (ORCID: https://orcid.org/0000-0003-2022-3669)
- Siân E. Handley (ORCID: https://orcid.org/0000-0002-7612-3889)
- Elisabeth De Smit (ORCID: https://orcid.org/0000-0002-3122-3122)
- Robert Henderson (ORCID: https://orcid.org/0000-0002-5250-6404)
- D M Thompson
- Weijen Tan
Institutions
- Great Ormond Street Hospital for Children NHS Foundation Trust (GB)
- University College London (GB)
Publication Details
- Journal
- JAMA Ophthalmology
- Published
- 2026-01-02
- DOI
- https://doi.org/10.1001/jamaophthalmol.2025.5505
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00