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Nat Med. 2023 Oct;29(10):2464-2472. doi: 10.1038/s41591-023-02520-3. Epub 2023 Oct 9.
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Full-field stimulus threshold testing: a scoping review of current practice.全场刺激阈值测试：当前实践的范围综述。
Eye (Lond). 2024 Jan;38(1):33-53. doi: 10.1038/s41433-023-02636-3. Epub 2023 Jul 13.
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Optimization and Validation of a Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations.虚拟现实定向和移动测试在遗传性视网膜变性中的优化和验证。
Transl Vis Sci Technol. 2023 Jan 3;12(1):28. doi: 10.1167/tvst.12.1.28.
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Mobility Testing and Other Performance-Based Assessments of Functional Vision in Patients with Inherited Retinal Disease.遗传性视网膜疾病患者的功能性视力的活动能力测试和其他基于表现的评估。
Cold Spring Harb Perspect Med. 2023 Jun 1;13(6):a041299. doi: 10.1101/cshperspect.a041299.
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Beyond the NEI-VFQ: Recent Experience in the Development and Utilization of Patient-Reported Outcomes for Inherited Retinal Diseases.超越NEI-VFQ：遗传性视网膜疾病患者报告结局的开发与应用新进展
Cold Spring Harb Perspect Med. 2023 Mar 1;13(3):a041298. doi: 10.1101/cshperspect.a041298.
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Improving Interpretation of Evidence Relating to Quality of Life in Health Technology Assessments of Rare Disease Treatments.改善罕见病治疗健康技术评估中与生活质量相关证据的解读。
Patient. 2023 Jan;16(1):7-17. doi: 10.1007/s40271-022-00598-4. Epub 2022 Oct 10.
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第二届 RDH12 相关遗传性视网膜退行性变临床试验设计和结局测量全球科学会议报告

Report From the Second Global Scientific Conference on Clinical Trial Design and Outcome Measures for RDH12-Associated Inherited Retinal Degeneration.

机构信息

Eyes on the Future, London, UK.

Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

出版信息

Transl Vis Sci Technol. 2024 Aug 1;13(8):17. doi: 10.1167/tvst.13.8.17.

DOI:10.1167/tvst.13.8.17
PMID:39120885
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11318357/
Abstract

A multi-stakeholder, patient centric approach will be critical to the design of future successful clinical trials with outcome measures relevant to the RDH12-IRD population.

摘要

采取多方利益相关者、以患者为中心的方法对于未来成功的临床试验设计至关重要，这些临床试验的结果衡量标准需要与 RDH12-IRD 人群相关。