Unraveling the basis of white matter disease in mucopolysaccharidosis type I – a DTI study

Awardee: Alena Svatkova

Institution: University of Minnesota

Award Amount: $35,000

Funding Period: January 1, 2015 - December 31, 2015

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Galectin-1 protein therapy for the treatment for Laminin alpha2 related Congenital Muscular Dystrophy

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Targeting miR-155 as a therapy for mucolipidosis IV