Phenylbutyrate for STXBP1 Encephalopathy – A Pilot Study

MDBRSTXBP1
Written By Million Dollar Bike Ride

Awardee: Zachary Grinspan

Institution: Weill Cornell Medicine

Grant Amount: $69,588

Million Dollar Bike Ride
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Rapid and unbiased in vivo screen for lead compounds that rescue STXBP1 dysfunction and subsequent in vitro validation in human neurons
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Towards new therapeutic treatments for the hyperinsulinism/hyperammonemia syndrome (HI/HA)