Pilot study to develop a small molecule activator of TCF4 as a treatment for Pitt-Hopkins syndrome

MDBRPitt Hopkins

Feb 1

Written By Million Dollar Bike Ride

Awardee: Ben Philpot

Institution: University of North Carolina, Chapel Hill

Award Amount: $51,242

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

MDBRPitt Hopkins2018

Million Dollar Bike Ride

Tissue-specific and temporal reinstatement of Tcf4 function to treat Pitt-Hopkins Syndrome

Gene Replacement Therapy for APBD