title: FDA approves ultra-orphan drug on a 4-patient trial.
authors.forename: Asher
authors.initials: A
authors.lastname: Mullard
mesh_terms: D002986:Clinical Trials as Topic; D017277:Drug Approval; D009097:Multienzyme Complexes / Q000235:genetics*; D009962:Orotate Phosphoribosyltransferase / Q000235:genetics*; D009963:Orotic Acid / Q000652:urine*; D009964:Orotidine-5'-Phosphate Decarboxylase / Q000235:genetics*; D009965:Orphan Drug Production; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D009097:Multienzyme Complexes; D009963:Orotic Acid; C027157:uridine 5'-monophosphate synthase; D009962:Orotate Phosphoribosyltransferase; D009964:Orotidine-5'-Phosphate Decarboxylase