Author

Kirill Zavalin

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Open access Jul 2026

4-Phenylbutyrate Rescue in GABRA1 Variants Associated with Developmental Epileptic Encephalopathies: From Cell and Mouse Models to Humans

This study indicates that PBA is a promising treatment option for DEEs associated with GABRA1 mutations and proposes that PBA holds promise as a common medicine for multiple genetic neurologic disorders that share the proteostasis pathology with a broad clinical application in DEEs.

Ziang Song, Kirill Zavalin, W. Shen et al. · Cells · 0 citations