Britten-Jones, Alexis Ceecee;
Al-Khuzaei, Saoud;
Rizzi, Matteo;
Crossland, Michael D;
Rozanowska, Malgorzata B;
Mendes, Bernardo S;
de Guimaraes, Thales AC;
... Stargardt's Connected; + view all
(2025)
Stargardt's Connected Research Network Inaugural Meeting: Landscape Review and Horizon Scanning of Stargardt Disease.
Translational Vision Science & Technology
, 14
(9)
, Article 26. 10.1167/tvst.14.9.26.
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Abstract
PURPOSE: The purpose of this study was to update the recent progress in the diagnosis, management, and treatments for Stargardt disease. METHODS: On November 22, 2024, Stargardt's Connected held its inaugural meeting of their Research Network, attended by clinicians, researchers, industry partners, and patient representatives. This meeting aimed to provide an update on Stargardt disease management and research and develop a call to action for the wider community. The format was rapid-fire presentations followed by a roundtable discussion. This review presents the meeting proceeding, along with a summary of best up-to-date evidence and key calls to action. RESULTS: Topics included: (1) advances in the understanding of Stargardt disease: natural history, genetic basis, animal models, and emerging therapies; (2) supporting individuals with Stargardt disease: disease impact, technological advancements, and lifestyle modifications; and (3) advancing research through stakeholder engagement, research registries, and patient input. The network acknowledges the importance of collaboration among patients, clinicians, researchers, and industry to address critical gaps in the diagnosis, management, and treatment of Stargardt disease. Patient engagement was emphasized as being crucial for driving progress in the field. CONCLUSIONS: Recent years have seen significant progress in understanding and managing Stargardt disease. Key calls to action included the areas of improving genetic testing and counseling, advancing research registries, supporting research and clinical trials, growing multidisciplinary care, addressing lifestyle and dietary modifications, and enhancing technological support. TRANSLATIONAL RELEVANCE: This Stargardt's Connected Research Network initiative outlines a multistakeholder engagement model to discuss ongoing research and emerging treatments for Stargardt disease, a condition with promising therapeutic developments.
| Type: | Article |
|---|---|
| Title: | Stargardt's Connected Research Network Inaugural Meeting: Landscape Review and Horizon Scanning of Stargardt Disease |
| Location: | United States |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1167/tvst.14.9.26 |
| Publisher version: | https://doi.org/10.1167/tvst.14.9.26 |
| Language: | English |
| Additional information: | Copyright 2025 The Authors This work is licensed under a Creative Commons Attribution 4.0 International License. |
| Keywords: | Stargardt Disease, Humans, Animals, Macular Degeneration |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10214077 |
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