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find Keyword "Inherited retinal dystrophy" 1 results
  • Chinese expert consensus on the standardized application of multi-luminance mobility test in clinical trials for inherited retinal dystrophy

    With the advancement of research on rare ocular diseases such as inherited retinal dystrophy (IRD) has advanced in recent years, especially breakthroughs in therapeutic approaches represented by cell and gene therapy, potential intervention strategies have emerged for these conditions. Establishing standardized endpoints and evaluation methods for visual function in patients with IRD has become crucial for assessing disease progression, safety, and therapeutic efficacy of innovative treatments. Best corrected visual acuity (BCVA) is widely recognized as one of the primary endpoints for assessing visual function. However, for IRD patients with severe rod photoreceptor dysfunction, who often present with profound low vision or even legal blindness, the applicability of BCVA as a traditional visual function indicator is limited. The multi-luminance mobility test (MLMT) has emerged as a functional visual assessment tool that evaluates the ability to navigate obstacle courses under varying illumination levels. By establishing graded evaluation standards, MLMT objectively quantifies the impact of lighting conditions on patients’ nobility and spatial orientation, providing a novel quantitative tool for assessing visual function in IRD clinical trials. Currently, there is a lack of unified and standardized guidelines for the use of MLMT in China, posing challenges to its implementation in practical clinical research. To address this, Ocular Fundus Disease Society of Chinese Medical Association and Chinese Medical Doctor Association convened a multidisciplinary team comprising clinical experts in genetic retinal diseases, statisticians, and optical specialists to investigate the current applications and technical characteristics of MLMT, ultimately formulating consensus recommendations for its use as a clinical trial endpoint for IRD gene therapies. This consensus aims to provide a set of MLMT operating norms applicable to China's national conditions, guide clinical practice and research in ophthalmology and related disciplines, and promote the standardization process of IRD clinical trials in China, so as to better serve the IRD patient population and promote the development of related fields.

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