CNS disease severity assessment with quantitative neuroimaging

Awardee: Igor Nestrasil

Institution: University of Minnesota

Grant Amount: $64,645

Funding Period: February 1, 2022 - January 31, 2023


Summary:

This project aims to find the underpinnings of brain magnetic resonance imaging (MRI) clinical/radiological severity in mucopolysaccharidosis type I (MPS I) and relate these findings to quantitative MRI outcomes. These links may ease the process of the central nervous system (CNS) disease severity assessment in the clinical setting.

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Understanding the role of inflammation in MPSIIIA disease to identify therapeutic approaches for older patients without treatment options

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Rescue of STXBP1 encephalopathies with small molecules in mouse models in vivo