| Title: |
Stargardt’s Connected Research Network Inaugural Meeting: Landscape Review and Horizon Scanning of Stargardt Disease |
| Authors: |
Britten-Jones, AC; Al-Khuzaei, S; Rizzi, M; Crossland, MD; Rozanowska, MB; Mendes, BS; de Guimaraes, TAC; Varela, MD; Sumodhee, D; Woof, WA; Khan, S; Girach, A; Downes, SM; Cheetham, ME; Piccolo, D; Ayton, LN; Brandão, ML; Scholl, HPN; Skilton, A; Tailor, B; Michaelides, M; Pontikos, N |
| Publisher Information: |
The Association for Research in Vision and Ophthalmology |
| Publication Year: |
2025 |
| Collection: |
The University of Melbourne: Digital Repository |
| Description: |
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 collabora tion 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. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| ISSN: |
2164-2591 |
| Relation: |
pii: 2810840; https://hdl.handle.net/11343/362990 |
| Availability: |
https://hdl.handle.net/11343/362990 |
| Rights: |
https://creativecommons.org/licenses/by/4.0 ; CC BY |
| Accession Number: |
edsbas.5E666901 |
| Database: |
BASE |