Evidence map›Paper›PMID 28920007›Full record

ReviewTranslational vision science & technology2017

Towards Treatment of Stargardt Disease: Workshop Organized and Sponsored by the Foundation Fighting Blindness.

Avery E Sears, Paul S Bernstein, Artur V Cideciyan, Carel Hoyng, Peter Charbel Issa, Krzysztof Palczewski, Philip J Rosenfeld, SriniVas Sadda, Ulrich Schraermeyer, Janet R Sparrow and 2 more

Open access · goldAbstract readReview
In one paragraph

Review in Translational vision science & technology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
39citing papers in PubMed, 1 pooled it
3.4field-weighted citation impact, top 7% of its field
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

39 citing papers in PubMed, 1 synthesis or guideline pooled it, 65 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Review
  8. Advances and therapeutic opportunities in visual cycle modulation.Progress in retinal and eye research · 2025
    Review
  9. Vitamin A supply in the eye and establishment of the visual cycle.Current topics in developmental biology · 2025
    Review
  10. Article
  11. Review
  12. Article
  13. Article
  14. Chemiexcitation and melanin in photoreceptor disc turnover and prevention of macular degeneration.Proceedings of the National Academy of Sciences of the United States of America · 2023
    Article
  15. Article
  16. Review
  17. Article
  18. Evaluation of Local Rod and Cone Function in Stargardt Disease.Investigative ophthalmology & visual science · 2022
    Article
  19. Article
  20. A five-year follow-up ofMolecular vision · 2022
    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 at 10 institutions in 5 countries.

Avery E SearsDepartment of Pharmacology, School of Medicine, Cleveland Center for Membrane and Structural Biology, Case Western Reserve University, Cleveland, OH, USA.
Paul S BernsteinMoran Eye Center, University of Utah, Salt Lake City, UT, USA.
Artur V CideciyanDepartment of Ophthalmology, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Carel HoyngDepartment of Ophthalmology, Radboud University Medical Center, Nijmegen, Netherlands.
Peter Charbel IssaOxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, and the Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom.
Krzysztof PalczewskiDepartment of Pharmacology, School of Medicine, Cleveland Center for Membrane and Structural Biology, Case Western Reserve University, Cleveland, OH, USA.
Philip J RosenfeldDepartment of Ophthalmology, Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA.
SriniVas SaddaDoheny Image Reading Center, Doheny Eye Institute, Los Angeles, CA, USA.
Ulrich SchraermeyerInstitute of Ophthalmic Research, Centre for Ophthalmology, University of Tübingen, Tübingen, Germany.
Janet R SparrowEdward S. Harkness Eye Institute, Columbia University Medical Center, New York, NY, USA.
Ilyas WashingtonEdward S. Harkness Eye Institute, Columbia University Medical Center, New York, NY, USA.
Hendrik P N SchollDepartment of Ophthalmology, University of Basel, Basel, Switzerland.
Case Western Reserve University · USColumbia University Irving Medical Center · USDoheny Eye Institute · USJohns Hopkins University · USOxford University Hospitals NHS Trust · GBPenn Presbyterian Medical Center · USRadboud University Medical Center · NLSTZ eyetrial · DEUniversity of Miami · USUniversity of Utah · US

Funding

MEDICAL SCIENTIST TRAINING PROGRAMT32GM007250 · NIGMS · CASE WESTERN RESERVE UNIVERSITY · PI HUANG, ALEX YEE-CHEN · 1985 to 2023
$33.4M
STRUCTURAL STUDIES OF ARRESTINSR01EY009339 · NEI · UNIVERSITY OF WASHINGTON · PI KISER, PHILIP DAVID, PALCZEWSKI, KRZYSZTOF · 1992 to 2025
$16.0M
University of Utah, Core Vision Research GrantP30EY014800 · NEI · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI PAUL STEVEN BERNSTEIN · 2005 to 2026
$14.6M
Instrumentation, Fabrication, and Design CoreP30EY019007 · NEI · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI Xin Zhang · 2010 to 2026
$12.9M
Use of systems pharmacology to prevent rod and cone photoreceptor degenerationR24EY027283 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI PALCZEWSKI, KRZYSZTOF · 2017 to 2021
$7.9M
Vision Research Training Grant at the University of UtahT32EY024234 · NEI · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI DAVID KRIZAJ, Monica L Vetter · 2014 to 2026
$2.3M
NEI NIH HHS P30 EY014800NEI NIH HHS P30 EY019007NEI NIH HHS R01 EY009339NEI NIH HHS R24 EY027283NEI NIH HHS T32 EY024234NIGMS NIH HHS T32 GM007250
6 · The paper itself

Abstract

Accumulation of fluorescent metabolic byproducts of the visual (retinoid) cycle is associated with photoreceptor and retinal pigment epithelial cell death in both Stargardt disease and atrophic (nonneovascular) age-related macular degeneration (AMD). As a consequence of this observation, small molecular inhibitors of enzymes in the visual cycle were recently tested in clinical trials as a strategy to protect the retina and retinal pigment epithelium in patients with atrophic AMD. To address the clinical translational needs for therapies aimed at both diseases, a workshop organized by the Foundation Fighting Blindness was hosted by the Department of Pharmacology at Case Western Reserve University on February 17, 2017, at the Tinkham Veale University Center, Cleveland, OH, USA. Invited speakers highlighted recent advances in the understanding of the pathophysiology of Stargardt disease, in terms of its clinical characterization and the development of endpoints for clinical trials, and discussed the comparability of therapeutic strategies between atrophic age-related macular degeneration (AMD) and Stargardt disease. Investigators speculated that reducing the concentrations of visual cycle precursor substances and/or their byproducts may provide valid therapeutic options for the treatment of Stargardt disease. Here we review the workshop's presentations in the context of published literature to help shape the aims of ongoing research endeavors and aid the development of therapies for Stargardt disease.

Indexed as

A2E, all-trans-retinalABCA4age-related macular degenerationgeographic atrophylipofuscinStargardt disease

Identifiers

PMID28920007
PMCPMC5599228
OpenAlexW2755243141

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.