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Author

M. V. van Muilekom

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Open access Sep 2026

Drug repurposing of glycerol phenylbutyrate for children with SLC6A1-related neurodevelopmental disorder: A prospective open-label case series.

Glycerol phenylbutyrate was associated with improvements in individualized outcomes in children with SLC6A1-NDD, however, personalized goal attainment measures are susceptible to expectation and observer bias in open-label study designs.

E. V. van Walree, D. Dekker, J. Sprengers et al. · 0 citations

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