Expanding the Breadth of Metabolite Analysis for Snyder Robinson Syndrome

Awardee: Tim Wood

Institution: Greenwood Genetic Center

Award Amount: $54,774

Funding Period: February 1, 2019 - January 31, 2020

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Skeletal stem cells to treat the bone defects of patients with Snyder-Robinson Syndrome

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Preclinical studies of FDA-approved small-molecules implementing lysosomal activity in a mouse model of MPSIIIA