Repurposing FDA-approved drugs for Niemann Pick C Disease

MDBRNPC
Written By Million Dollar Bike Ride

Awardee: Yamuna Krishnan

Institution: University of Chicago

Award Amount: $62,340

Million Dollar Bike Ride
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Advancing A Novel Therapeutic Lead for RASopathies
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Muscle Directed Gene Therapy Application for Maple Syrup Urine Disease (MSUD)