Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model

MDBRMSUD
Written By Million Dollar Bike Ride

Awardee: Rebecca Ahrens-Nicklas

Institution: The Children's Hospital of Philadelphia / The University of Pennsylvania

Award Amount: $44,037

Million Dollar Bike Ride
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A comparative and quantitative approach to study lysosomal dysfunction in Mucopolysaccharidoses diseases
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Development of novel human stem cell models of Beta-Propeller Protein- Associated Neurodegeneration (BPAN) for disease modelling and drug screening