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Clinical Trials for Metal Metabolism, Inborn Errors

• Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
• Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
• Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
• Apotransferrin in Atransferrinemia
• Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A