Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease.

MDBRNUBPL

Feb 1

Written By Million Dollar Bike Ride

Awardee: Marni Falk

Institution: Children's Hospital of Philadelphia and University of Pennsylvania

Award Amount: $61,134

MDBRNUBPL2019

Million Dollar Bike Ride

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

Genetic Mechanisms in Neuroendocrine cell Hyperplasia of Infancy (NEHI)