ArticleTranslational vision science & technology2026
Voretigene Neparvovec Gene Therapy in Clinical Practice: A 12-Month, Single-Center, In-Depth Analysis of Beneficial and Adverse Drug Effects.
Article in Translational vision science & technology, 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
11 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: To investigate the effects of voretigene neparvovec (VN) treatment in patients with RPE65-related retinal dystrophies, with particular focus on chorioretinal atrophy (CRA) and its impact on visual function. Methods: Twenty-five patients treated with VN were evaluated over a 12-month follow-up and stratified according to the presence of postoperative CRA, classified into nummular, mixed and touchdown patterns. Results: Best-corrected visual acuity (BCVA) improved by a median of 11 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (P < 0.001). Semiautomated kinetic visual field (SKVF) showed a significant (P < 0.001) enlargement for both I4e and III4e stimulus sizes. Fullfield stimulus threshold (FST) results also improved significantly (P < 0.001), with median changes of -2.79 (-3.61 to -2.11) log cd·s/m2 for white, -3.02 (-4.05 to -2.24) log cd·s/m2 for blue, and -1.30 (-1.76 to -0.74) log cd·s/m2 for red stimuli. Fifteen patients developed CRA (60%), of whom nine exhibited progressive nummular/mixed CRA, which significantly expanded over time (P < 0.001), and six showed the localized, non-progressive touchdown pattern. Younger age and a clinical diagnosis of LCA were significantly associated with nummular/mixed CRA. No significant differences in BCVA or FST improvements were found between nummular/mixed and touchdown/no-CRA groups. Conversely, significant SKVF I4e enlargement occurred only in the touchdown/no-CRA subgroup (1624°2, 0-6278; P < 0.001), with a significant intergroup difference (P = 0.003). Conclusions: Our findings confirmed the visual function improvements following VN and the relatively high prevalence of CRA, which seems to impact negatively only on SKVF enlargement. Translational Relevance: These findings are useful to better understand risks and benefits of VN gene therapy.
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.