Article

Virtual screening and zebrafish phenotype-based evaluation argues against repurposing 4-phenylbutyrate for STXBP1-related disorders

2026-05-12

Abstract excerpt

Syntaxin-binding protein 1 ( STXBP1 ) mutations lead to severe epilepsy, intellectual disability, developmental delay, and movement disorder. Effective treatments for these conditions do not exist. Recent studies in Munc18-1 ( STXBP1 ) C. elegans models demonstrate that 4-phenylbutyrate (4-PBA) or related pharmacological chaperones stabilize Munc18-1 protein levels and rescue locomotion deficits. These studies...

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Literature Corpus work: 3bbc4362-b424-57c4-90fc-f674614541ec
DOI: 10.64898/2026.05.07.723632

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