Reverse amblyopia following gene therapy-induced visual improvement in a child with retinal pigment epithelium-65 inherited retinal dystrophy.

Authors

Aimee Lloyd, Peter Kiraly, Evita Evangelia Christou, Jane Ashworth, Assad Jalil, Tsveta Ivanova

Published in

BMJ case reports. Volume 19. Issue 8. Aug 20, 2026. Epub Aug 20, 2026.

Abstract

Voretigene neparvovec can improve visual function in children with RPE65-associated inherited retinal dystrophy. However, the effect of rapid asymmetric visual improvement on binocular development and amblyopia remains uncertain.A young child underwent sequential treatment 8 months apart. After right-eye treatment, visual acuity improved, fixation switched and a manifest left esotropia developed. Left-eye visual acuity subsequently deteriorated. We describe this as reverse amblyopia in a novel context: reversal of the interocular amblyopic pattern following treatment-induced visual improvement without fellow-eye penalisation. Despite left-eye treatment, refractive correction and attempted occlusion, the marked visual asymmetry persisted. This case highlights a potential binocular effect of rapid unilateral visual improvement and the need for close monitoring between sequential treatments.

PMID:
42624634
Bibliographic data and abstract were imported from PubMed on 21 Aug 2026.

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