Alexis Ceecee Britten-Jones, Saoud Al-Khuzaei, Matteo Rizzi, Michael D Crossland, Malgorzata B Rozanowska, Bernardo S Mendes, Thales A C de Guimaraes, Malena Daich Varela, Dayyanah Sumodhee, William A Woof, Seemi Khan, Aniz Girach, Susan M Downes, Michael E Cheetham, Davide Piccolo, Lauren N Ayton, Marina Leite Brandão, Hendrick P N Scholl, Andi Skilton, Bhavna Tailor, Michel Michaelides, Nikolas Pontikos
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引用次数: 0
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.
期刊介绍：
Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO), an international organization whose purpose is to advance research worldwide into understanding the visual system and preventing, treating and curing its disorders, is an online, open access, peer-reviewed journal emphasizing multidisciplinary research that bridges the gap between basic research and clinical care. A highly qualified and diverse group of Associate Editors and Editorial Board Members is led by Editor-in-Chief Marco Zarbin, MD, PhD, FARVO.
The journal covers a broad spectrum of work, including but not limited to:
Applications of stem cell technology for regenerative medicine,
Development of new animal models of human diseases,
Tissue bioengineering,
Chemical engineering to improve virus-based gene delivery,
Nanotechnology for drug delivery,
Design and synthesis of artificial extracellular matrices,
Development of a true microsurgical operating environment,
Refining data analysis algorithms to improve in vivo imaging technology,
Results of Phase 1 clinical trials,
Reverse translational ("bedside to bench") research.
TVST seeks manuscripts from scientists and clinicians with diverse backgrounds ranging from basic chemistry to ophthalmic surgery that will advance or change the way we understand and/or treat vision-threatening diseases. TVST encourages the use of color, multimedia, hyperlinks, program code and other digital enhancements.
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