Weekly shot tested in babies with rare metabolic disorder
NCT ID NCT06582524
Summary
This study tested a weekly injection called pegzilarginase in babies under 2 years old with a rare genetic disorder called arginase 1 deficiency. The goal was to see if the treatment safely lowers high levels of a harmful substance called arginine in the blood. Three very young children received the injections for 12 weeks while researchers monitored their safety and blood levels.
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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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