ArticleEye (London, England)2025
Voretigene neparvovec in RPE65-related inherited retinal dystrophy: the 1-year real-world study LIGHT.
Article in Eye (London, England), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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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.
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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.
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Who cites it
3 citing papers in PubMed.
- Clinical and pharmacovigilance safety evaluation of LUXTURNACutaneous and ocular toxicology · 2025Review
- Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium.Translational vision science & technology · 2025Review
- In Vivo Cone Photoreceptor Topography of the Human Foveola.Investigative ophthalmology & visual science · 2025Article
Corrections and comments
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Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThis retrospective real-world study evaluated the effectiveness and safety of subretinal voretigene neparvovec (VN) in French patients (six children, six adults) with inherited retinal dystrophies.
methodsData were collected from medical records for the year following bilateral treatment with subretinal VN. Functional vision was assessed using the Streetlab mobility course with obstacles. The main outcome was Percentage of Preferred Walking Speed (PPWS) 1 month after treatment (the average speed to navigate the obstacle course as a percentage of the speed to walk straight for 4 m without obstacles at 500 lux).
resultsPPWS median relative improvement to month 1 was 63.9% (interquartile range 47.8%; 88.5%) at 2 lux. Gains were sustained to month 6, were comparable in children and adults, with similar patterns at 7.5 and 50 lux. Course completion time and the number of collisions improved at month 1 at 2 lux. Median full-field stimulus test (FST) improved at month 1 (-33.2 [interquartile range -33.7; -19.8] dB), with improvements sustained to month 24. Over the study, best corrected visual acuity (BCVA) appeared stable, and kinetic visual field had no discernible pattern. All patients experienced at least one ocular AE related to surgery, VN, or both. One serious AE occurred (retinal detachment in a child), and was considered related to surgery. Chorioretinal atrophies occurred in four adults and one child, with no impact on FST.
conclusionsFunctional vision, measured in the Streetlab mobility course, demonstrated rapid, sustainable improvements in lower light intensities. Safety data were in line with current knowledge for VN.
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