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

MDBRMSUD

Feb 1

Written By Million Dollar Bike Ride

Awardee: Jenny Greig

Institution: Gene Therapy Program, University of Pennsylvania

Award Amount: $112,893

Funding Period: February 1, 2019 - January 31, 2020

MDBRMSUD2018

Million Dollar Bike Ride

Repurposing FDA-approved drugs for Niemann Pick C Disease

TFEB and Lysosomal exocytosis to prevent lung destruction in LAM