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

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Title: Impact of glycerol phenylbutyrate on biochemistry and outcomes in paediatric patients with urea cycle disorders: a multicentre case series from Saudi Arabia.
Authors: Hejazi R; Alfaisal University, Riyadh, Saudi Arabia., Alghamdi TH; King Abdulaziz Medical City, King Abdullah Specialized Children Hospital, Ministry of National Guard-Health Affairs (NGHA), Riyadh, Saudi Arabia., Salih R; Prince Sultan Military Medical City, Riyadh, Saudi Arabia., Naeim Y; Prince Sultan Military Medical City, Riyadh, Saudi Arabia., Althiyab H; King Abdulaziz Medical City, King Abdullah Specialized Children Hospital, Ministry of National Guard-Health Affairs (NGHA), Riyadh, Saudi Arabia., AlAnzi T; Johns Hopkins Aramco Healthcare Centre, Dhahran, Saudi Arabia., Mohamed S; Alfaisal University, Riyadh, Saudi Arabia.; Prince Sultan Military Medical City, Riyadh, Saudi Arabia.; National University, Khartoum, Sudan., Alfadhel M; King Abdulaziz Medical City, King Abdullah Specialized Children Hospital, Ministry of National Guard-Health Affairs (NGHA), Riyadh, Saudi Arabia.; King Saud bin Abdulaziz University for Health Sciences, Ministry of National Guard-Health Affairs (NGHA), Riyadh, Saudi Arabia.; King Abdullah International Medical Research Centre (KAIMRC), Riyadh, Saudi Arabia., ALTassan RS; Alfaisal University, Riyadh, Saudi Arabia.; King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia., Al-Hassnan ZN; Alfaisal University, Riyadh, Saudi Arabia.; King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia., AlSayed M; Alfaisal University, Riyadh, Saudi Arabia. moeen@kfshrc.edu.sa.; King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. moeen@kfshrc.edu.sa.
Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2026 Jan 30; Vol. 21 (1). Date of Electronic Publication: 2026 Jan 30.
Publication Type: Journal Article; Multicenter Study
Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1750-1172
DOI:10.1186/s13023-026-04216-6