Evidence map›Paper›PMID 41140124›Full record

ReviewCutaneous and ocular toxicology2025

Clinical and pharmacovigilance safety evaluation of LUXTURNA

Djamilla Simoens, Varun Shravah, Walter Keith Jones, Simon Kaja

Abstract readReview
In one paragraph

Review in Cutaneous and ocular toxicology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. 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

4 authors.

Djamilla SimoensProgram in Pharmacovigilance, Loyola University Chicago, Health Sciences Campus, Maywood, IL, USA.
Varun ShravahStritch School of Medicine, Loyola University Chicago, Health Sciences Campus, Maywood, IL, USA.
Walter Keith JonesProgram in Pharmacovigilance, Loyola University Chicago, Health Sciences Campus, Maywood, IL, USA.
Simon KajaProgram in Pharmacovigilance, Loyola University Chicago, Health Sciences Campus, Maywood, IL, USA.

Funding

Immunotherapy for Ocular Surface DiseasesR24EY032440 · NEI · UNIVERSITY OF ILLINOIS AT CHICAGO · PI JAIN, SANDEEP · 2021 to 2025
$8.3M
Basic and Clinical Research Training for Medical StudentsT35HL120835 · NHLBI · LOYOLA UNIVERSITY CHICAGO · PI ZELEZNIK-LE, NANCY J · 2015 to 2024
$628k
NEI NIH HHS R24 EY032440NHLBI NIH HHS T35 HL120835
6 · The paper itself

Abstract

backgroundLUXTURNA

methodsWe reviewed published literature and regulatory safety databases, including the FDA Adverse Event Reporting System and EudraVigilance, to assess clinical outcomes and adverse event reporting associated with LUXTURNA

resultsClinical trials demonstrated durable functional vision improvements, with approximately 70% of patients maintaining gains up to four years post-treatment. Adverse events reported in trials were largely mild to moderate, such as conjunctival hyperemia, cataract, and transient intraocular pressure elevation. Post-marketing surveillance, however, revealed additional safety concerns, most notably chorioretinal atrophy (CRA), which has been reported in 13-50% of treated eyes, particularly in younger patients and often near the injection site. Disproportionality analyses confirmed CRA as the most prominent adverse event, alongside elevated risks for retinal tears or detachment, intraocular pressure changes, ocular hemorrhage, and inflammation. Despite these findings, central visual acuity was generally preserved, and no systemic toxicity was identified.

conclusionsLUXTURNA

Indexed as

Genetic Therapycis-trans-IsomerasesHumansPharmacovigilanceRetinoid Isomerohydrolasecis-trans-IsomerasesRetinoid IsomerohydrolaseFAERS databaseGene therapyinherited retinal diseaseLeber’s congenital amaurosisLuxturnaocular safetypharmacovigilanceretinitis pigmentosaRPE65voretigene neparvovec

Identifiers

PMID41140124
PMCPMC12616891

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

None linked

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.