title: Setting up an emergency stock for metabolic diseases.
abstract: BACKGROUND
  Therapeutic management of inborn errors of metabolism (IEMs) is complicated. The drugs involved are classified as orphan, and their supply depends on whether they are orphan medicines, investigational drugs, or need to be prepared as a compounded formula.
  
  
  METHODS
  We analyzed emergency criteria, availability, and permanent location of metabolic drugs within the hospital. Information on therapeutic usage, administration, and dosage was also recorded.
  
  
  RESULTS
  A stock for treating IEMs should include chelating agents, drugs to treat deficiencies, enzyme supplements, and other specific treatments. Hyperammonemia was considered to be life-threatening; therefore, an emergency supply of drugs to treat this condition should be kept permanently in the hospitalization unit.
  
  
  CONCLUSIONS
  Emergency drug stocks are highly recommended in tertiary hospitals in order to improve care for patients susceptible to IEM.
authors.affiliation: Pharmacy Department, Hospital General Universitario Gregorio Marañon, C/ Dr. Esquerdo 46, Madrid, Spain. cmartinezf.hgugm@salud.madrid.org
authors.forename: C M
authors.initials: CM
authors.lastname: Fernandez-Llamazares
authors.forename: M L
authors.initials: ML
authors.lastname: Serrano
authors.forename: S
authors.initials: S
authors.lastname: Manrique-Rodríguez
authors.forename: M
authors.initials: M
authors.lastname: Sanjurjo-Sáez
mesh_terms: D000138:Acidosis / Q000188:drug therapy; D000596:Amino Acids / Q000600:supply & distribution / Q000627:therapeutic use; D002331:Carnitine / Q000600:supply & distribution / Q000627:therapeutic use; D002614:Chelating Agents / Q000600:supply & distribution / Q000627:therapeutic use; D019468:Disease Management; D055695:Electronic Prescribing; D004630:Emergencies; D057487:Enzyme Therapy; D004798:Enzymes / Q000600:supply & distribution; D006297:Health Services Accessibility; D006748:Hospital Departments; D006771:Hospitals, Maternity; D006776:Hospitals, Pediatric; D006801:Humans; D022124:Hyperammonemia / Q000188:drug therapy; D007445:Inventories, Hospital / Q000458:organization & administration*; D008510:Medication Systems, Hospital / Q000458:organization & administration*; D008661:Metabolism, Inborn Errors / Q000188:drug therapy* / Q000453:epidemiology; D009965:Orphan Drug Production; D010607:Pharmacy Service, Hospital / Q000331:legislation & jurisprudence / Q000458:organization & administration*; D013030:Spain
chemical_list: D000596:Amino Acids; D002614:Chelating Agents; D004798:Enzymes; D002331:Carnitine