Towards new therapeutic treatments for the hyperinsulinism/hyperammonemia syndrome (HI/HA)

MDBRCHI

Feb 1

Written By Million Dollar Bike Ride

Awardee: Thomas Smith

Institution: University of Texas Medical Branch

Grant Amount: $72,014

MDBRCHI2019

Million Dollar Bike Ride

Phenylbutyrate for STXBP1 Encephalopathy – A Pilot Study

What are the functional consequences of CDKL5 dysfunction in human neuronal cells?