While research has accelerated the development of new treatments for pediatric neurodegenerative 20 disorders, the ability to demonstrate the long-term efficacy of these therapies has been hindered by the lack of 21 convincing, noninvasive methods for tracking disease progression both in animal models and in human clinical 22 trials. Here, we unveil a new translational platform for tracking disease progression in an animal model of a 23 pediatric neurodegenerative disorder, CLN6-Batten disease. Instead of looking at a handful of parameters or a single 24 "needle in a haystack", we embrace the idea that disease progression, in mice and patients alike, is a diverse 25 phenomenon best characterized by a combination of relevant biomarkers. Thus, we employed a multi-modal 26 quantitative approach where 144 parameters were longitudinally monitored to allow for individual variability. We 27 use a range of noninvasive neuroimaging modalities and kinematic gait analysis, all methods that parallel those 28 commonly used in the clinic, followed by a powerful statistical platform to identify key progressive anatomical and 29 metabolic changes that correlate strongly with the progression of pathological and behavioral deficits. This 30 innovative, highly sensitive platform can be used as a powerful tool for preclinical studies on neurodegenerative 31 diseases, and provides proof-of-principle for use as a potentially translatable tool for clinicians in the future. 32 33 [Main Text: ] 34
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