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Article

Differential Tractography: A Biomarker for Neuronal Function in Neurodegenerative Disease

2024-08-26

Abstract excerpt

GM1 gangliosidosis is an ultra-rare inherited neurodegenerative lysosomal storage disorder caused by biallelic mutations in the GLB1 gene. GM1 is uniformly fatal and has no approved therapies, although clinical trials investigating gene therapy as a potential treatment for this condition are underway. Novel outcome measures or biomarkers demonstrating the longitudinal effects of GM1 and potential recovery due to t...

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Literature Corpus work
3c8cf258-efc9-5010-90e9-a39eea8ce20c
DOI
10.1101/2024.08.25.24312255
Open publication

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Differential Tractography: A Biomarker for Neuronal Function in Neurodegenerative DiseaseDOI 10.1101/2024.08.25.24312255
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