Brain MRI signatures in infants with infantile forms of GM-1 and GM-2

2017NTSAD

Jan 1

Written By Million Dollar Bike Ride

Awardee: Igor Nestrasil

Institution: University of Minnesota

Award Amount: $42,419

Funding Period: January 1, 2018 - December 31, 2018

MDBR2017NTSAD

Million Dollar Bike Ride

Mechanisms of mitochondrial toxicity in Snyder-Robinson Syndrome

Pilot study to identify small molecule activators of TCF4 as a treatment for Pitt-Hopkins syndrome