New enzyme therapy shows promise for rare metabolic disorder in babies
NCT ID NCT06582524
First seen Jan 11, 2026 · Last updated May 23, 2026 · Updated 12 times
Summary
This study tested a weekly injection of pegzilarginase in 3 infants under 2 years old with Arginase 1 Deficiency, a rare genetic disorder that causes harmful buildup of arginine. The goal was to see if the drug safely lowers arginine levels and helps manage the condition. Participants received treatment for 12 weeks, followed by 8 weeks of safety monitoring.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Bradford Royal Infirmary Duckworth Lane
Bradford, BD9 6RJ, United Kingdom
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Unidade de Doenças Metabólicas Pediatria, Hospital Santa Maria
Lisbon, Portugal
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Univ. Klinik für Kinder- und Jugendheilkunde Medizinische Universität
Graz, A-8036, Austria
Conditions
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