Neurocognitive Outcomes and Quality of Life in Adults with MSUD

Awardee: Jessica Gold

Institution: Children's Hospital of Philadelphia

Award Amount: $54,465

Funding Period: February 1, 2021 - January 31, 2022

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Deciphering the causes of mitochondrial network disruption in WDR45-defective cells and their contribution to the BPAN pathology

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Tissue inflammation in Mucopolysaccharidoses type II disease: molecular mechanism and therapy