Rapid and unbiased in vivo screen for lead compounds that rescue STXBP1 dysfunction and subsequent in vitro validation in human neurons

MDBRSTXBP1
Written By Million Dollar Bike Ride

Awardee: Matthijs Verhage

Institution: Rapid and unbiased in vivo screen for lead compounds that rescue STXBP1 dysfunction and subsequent in vitro validation in human neurons

Grant Amount: $69,588

Million Dollar Bike Ride
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Dimethylspermine (Me2Spm) as a novel treatment approach to restore normal spermidine/spermine ratios in Snyder-Robinson Syndrome (SRS) models
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Phenylbutyrate for STXBP1 Encephalopathy – A Pilot Study