Records containing: Molybdenum Cofactor Deficiency, Type A
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CLINICAL_TRIALS study record update · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
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CLINICAL_TRIALS study record update · 2023-10-17A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
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CLINICAL_TRIALS study record update · 2023-10-17A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A