Therapy of X-linked adrenoleukodystrophy

NeuroRx. 2006 Apr;3(2):246-53. doi: 10.1016/j.nurx.2006.01.004.

Abstract

Current therapies for X-linked adrenoleukodystrophy (X-ALD) include replacement therapy with adrenal steroids, which is mandatory for all patients with impaired adrenal function but does not alter neurological progression significantly; dietary therapy with "Lorenzo's Oil," which appears to have a preventive effect in asymptomatic boys whose brain MRI is normal; and hematopoietic stem cell transplantation in patients in the early stage of the cerebral inflammatory phenotype. Application of these interventions requires careful assessment of the patients' phenotype, which often changes over time. Family screening provides important opportunities for disease prevention.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Adrenoleukodystrophy / diet therapy
  • Adrenoleukodystrophy / genetics
  • Adrenoleukodystrophy / surgery
  • Adrenoleukodystrophy / therapy*
  • Drug Combinations
  • Erucic Acids / therapeutic use
  • Hematopoietic Stem Cell Transplantation
  • Hormone Replacement Therapy
  • Humans
  • Phenotype
  • Triolein / therapeutic use

Substances

  • Drug Combinations
  • Erucic Acids
  • Lorenzo's oil
  • Triolein