Repurposing FDA-approved drugs for Niemann Pick C Disease

MDBRNPC

Feb 1

Written By Million Dollar Bike Ride

Awardee: Yamuna Krishnan

Institution: University of Chicago

Award Amount: $62,340

MDBRNPC2018

Million Dollar Bike Ride

Advancing A Novel Therapeutic Lead for RASopathies

Muscle Directed Gene Therapy Application for Maple Syrup Urine Disease (MSUD)