Impact of glycerol phenylbutyrate on biochemistry and outcomes in paediatric patients with urea cycle disorders: a multicentre case series from Saudi Arabia.

Orphanet J Rare Dis · 2026 · PMC12934098 · PMID 41618427

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did not reach statistical significanceno p-value reported

Annualised HAC rates fell by 55% (2.2 to 1.0/year), HAC-related hospitalisations by 27% (1.1 to 0.8/year), and annualised HAC-related hospital stays by 24% (3.3 to 2.5 days/year), though these did not reach statistical significance.

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