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Virtual Screening and Zebrafish Phenotype‐Based Evaluation Argues Against Repurposing 4‐Phenylbutyrate for STXBP1‐Related Disorders

ABSTRACT 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)...

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Bibliografiska uppgifter
Huvudupphov: Aline Frick, Paige Whyte‐Fagundes, Scott C. Baraban
Materialtyp: Artigo
Språk:Inglês
Utgiven: Wiley 2026-06-01
Serie:Pharmacology Research & Perspectives
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Länkar:https://doi.org/10.1002/prp2.70285
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