Journal of Inherited Metabolic Disease

, Volume 18, Issue 4, pp 435–447

Adrenoleukodystrophy: natural history, treatment and outcome

  • H. W. Moser
Komrower Lecture

DOI: 10.1007/BF00710055

Cite this article as:
Moser, H.W. J Inherit Metab Dis (1995) 18: 435. doi:10.1007/BF00710055

Summary

Our laboratory has identified nearly 2000 patients with X-linked adrenoleukodystrophy (ALD) and conducted therapeutic trials in groups of patients who represent the major phenotypes. We report recent results of dietary therapy with a mixture of glyceryl trioleate and glyceryl trierucate oil, also referred to as Lorenzo's Oil, in the asymptomatic and childhood cerebral phenotypes. Fifty-three patients started this therapy at a mean age of 7.5 years at a time when they were free of neurological symptoms. Although analysis of data is hampered by the lack of a concurrent control group, follow-up studies after 39 months of therapy suggest that subsequent neurological involvement was less frequent and less severe than anticipated from historical controls. Retrospective analysis of the effect of the oil in patients with the severe childhood cerebral phenotype indicates that there was a slight but statistically significant slowing of clinical progression and delay of death.

Copyright information

© SSIEM and Kluwer Academic Publishers 1995

Authors and Affiliations

  • H. W. Moser
    • 1
  1. 1.Kennedy Krieger Institute, and the Departments of Neurology and PediatricsJohns Hopkins UniversityBaltimoreUSA