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

Awardee: Ben Philpot

Institution: University of North Carolina, Chapel Hill

Award Amount: $51,242

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

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

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Gene Replacement Therapy for APBD