ArticleKorean journal of ophthalmology : KJO2026
Four-Year Structural and Functional Outcomes of the First Subretinal Voretigene Neparvovec-rzyl Treatment for RPE65-Associated Inherited Retinal Dystrophy in South Korea.
Article in Korean journal of ophthalmology : KJO, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
purposeTo describe 4-year functional and structural outcomes of the first bilateral subretinal voretigene neparvovec-rzyl (VN) in a South Korean patient with molecularly confirmed RPE65-associated inherited retinal dystrophy, with a focus on the relationship between chorioretinal atrophy and visual function.
methodsThis was an observational case-based study of a 29-year-old female patient with biallelic RPE65 pathogenic variants. Both eyes underwent 25-gauge pars plana vitrectomy and subretinal VN injection 7 days apart. Standardized assessments were performed at baseline and 1 month, 3 months, 6 months, 1 year, 2 years, 3 years, and 4 years. Functional outcomes included best-corrected visual acuity (BCVA; Early Treatment Diabetic Retinopathy Study [ETDRS] letters), kinetic visual fields, full-field stimulus threshold (FST) testing with white-light, and multi-luminance mobility test (MLMT). Structural changes were evaluated with spectral-domain optical coherence tomography, ultra-widefield color and autofluorescence imaging, and infrared reflectance.
resultsAt baseline, BCVA was 9 and 8 ETDRS letters (right/left), with widespread ellipsoid zone loss but relatively preserved retinal pigment epithelium. BCVA improved to 34 / 33 letters at 6 months and remained higher than baseline in both eyes at 4 years. Goldmann III4e average visual field radius increased from 2.1° / 0.3° at baseline to 8.8° / 15.0° at 3 months, followed by partial contraction with paracentral scotomas after 1 year, yet fields remained wider than baseline at 4 years. FST improved from 0.10 log10(cd·s/m²) to -1.46 log10(cd·s/m²) at 1 month and remained stable at 3 months. MLMT performance improved from 150 lux at baseline to 10 lux binocularly and was maintained at 4 years. Perifoveal, fovea-involving chorioretinal atrophy developed between 3 and 6 months and enlarged mainly during the first year, then progressed slowly thereafter.
conclusionsIn this South Korean patient, VN provided large and durable functional gains over 4 years despite early, severe chorioretinal atrophy, illustrating structural-functional dissociation and underscoring the need for long-term multimodal monitoring in RPE65-associated disease.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.