Carbamoyl Phosphate Synthetase Deficiency
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CLINICAL_TRIALS trial_result · 2025-07-20Clinical Trial Update: Hepatic Histopathology in Urea Cycle Disorders
This clinical trial update indicates the study on hepatic histopathology in urea cycle disorders is actively recruiting, including Ornithine…
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EMA approval · 2025-01-14EMA Authorised: Pheburane (sodium phenylbutyrate) — Eurocept International B. V.
The EMA authorised Pheburane (sodium phenylbutyrate) for treating carbamoyl-phosphate synthetase I deficiency, with decision on 17 December …
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CLINICAL_TRIALS withdrawal · 2014-05-29Clinical Trial Update: Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate [Phase 2]
Phase 2 trial of N-Carbamylglutamate to increase ureagenesis in urea cycle disorders and other inborn errors of metabolism has been withdraw…