Evidence map›Paper›PMID 41853567›Full record

ArticleOphthalmology science2026

Expert Consensus on Characteristics, Etiology, and Management of Chorioretinal Atrophy in Patients Treated with Voretigene Neparvovec.

M Dominik Fischer, Isabelle Audo, David Gaucher, Frank G Holz, Line Kessel, Stephen Russell, Katarina Stingl, David L Rousso, Rainer Maier, Andreas Clemens and 2 more

Abstract read
In one paragraph

Article in Ophthalmology science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

M Dominik FischerOxford Eye Hospital, University of Oxford NHS Foundation Trust and NIHR Oxford Biomedical Research Centre, Oxford, UK.
Isabelle AudoSorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France.
David GaucherDepartment of Ophthalmology, Nouvel Hospital Civil, Strasbourg University Hospital, FMTS, University of Strasbourg, Strasbourg, France.
Frank G HolzDepartment of Ophthalmology, University of Bonn, Bonn, Germany.
Line KesselDepartment of Ophthalmology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
Stephen RussellThe University of Iowa Institute for Vision Research, University of Iowa, Iowa City, Iowa.
Katarina StinglUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
David L RoussoSpark Therapeutics, Inc., Philadelphia, Pennsylvania.
Rainer MaierNovartis Pharma AG, Basel, Switzerland.
Andreas ClemensNovartis Pharma AG, Basel, Switzerland.
Aaron NagielRoski Eye Institute, Department of Ophthalmology, Keck School of Medicine, University of Southern California, Los Angeles, California.
Bart P LeroyDepartment of Ophthalmology & Centre for Medical Genetics, Ghent University Hospital & Ghent University, Ghent, Belgium.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Purpose: Voretigene neparvovec (VN), developed for the treatment of inherited retinal dystrophy (IRD) associated with confirmed biallelic Design: Review of the scientific literature and expert consensus. Participants: An international group of experts in IRD with experience treating with VN. Methods: A literature search of PubMed was performed using broad search terms to return all publications relating to VN, which were manually screened for relevance to CRA. The expert panel discussed the literature and proposed updated consensus nomenclature and suggestions for monitoring. Results: According to most reports, development of CRA does not impact visual and functional outcomes after subretinal VN injection. Longer follow-up is needed to ascertain whether CRA continues to enlarge and whether treatment outcomes can be maintained. Chorioretinal atrophy growth might be caused by a combination of multiple factors, and several hypotheses on CRA etiology based on published clinical evidence are discussed. Standard terminology and metrics for CRA would support these future research efforts. We propose using the term "injection site CRA" to refer to CRA which develops specifically at the location where the cannula touches down onto the retina. For all other CRA, we propose defining atrophy based on its retinal localization, as either "central CRA" or "peripheral CRA." Regular follow-up visits using a combination of imaging modalities with efficacy readouts are recommended to track efficacy and adverse outcomes, including CRA. Conclusions: Numerous questions remain regarding causes of CRA in Financial Disclosures: Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

Indexed as

Chorioretinal atrophyGene therapyInherited retinal dystrophyRPE65Voretigene neparvovec

Identifiers

PMID41853567
PMCPMC12993124

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Registered trials

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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.