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

MDBRMSUD

Feb 1

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

MDBRMSUD2019

Million Dollar Bike Ride

A comparative and quantitative approach to study lysosomal dysfunction in Mucopolysaccharidoses diseases

Development of novel human stem cell models of Beta-Propeller Protein- Associated Neurodegeneration (BPAN) for disease modelling and drug screening