Urea Cycle Disorders Inborn
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency [Phase 1, Phase 2]
This clinical trial update describes an open-label Phase 1/2 study evaluating ECUR-506 in male infants under 9 months with neonatal-onset Or…
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CLINICAL_TRIALS trial_result · 2025-02-03Clinical Trial Update: Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Long-term follow-up study update for participants in any iECURE protocol using an investigational product; the trial remains in RECRUITING s…
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EMA withdrawal · 2018-07-05EMA Application withdrawn: Prohippur (sodium benzoate) — Lucane Pharma
The EMA withdrawed the application for Prohippur (sodium benzoate) by Lucane Pharma for urea cycle disorders and hyperglycinemia, nonketotic…