Urea cycle defects: management and outcome

J Inherit Metab Dis. 2005;28(3):407-14. doi: 10.1007/s10545-005-0303-7.

Abstract

This paper reviews the clinical presentation of 217 patients with urea cycle defects, including 121 patients with neonatal-onset forms and 96 patients with late-onset forms. Long-term outcome of these patients is also reported with the severity of the neonatal forms of these disorders, mostly for ornithine carbamoyltransferase-deficient males. Patients with late-onset forms may present at any age and carry a 28% mortality rate and a subsequent risk of subsequent disabilities.

Publication types

  • Review

MeSH terms

  • Adolescent
  • Adult
  • Aging / physiology
  • Child
  • Child, Preschool
  • Humans
  • Infant
  • Infant, Newborn
  • Metabolism, Inborn Errors / diagnosis
  • Metabolism, Inborn Errors / epidemiology
  • Metabolism, Inborn Errors / therapy*
  • Retrospective Studies
  • Treatment Outcome
  • Urea / metabolism*

Substances

  • Urea