Experience on therapy of adrenoleukodystrophy and adrenomyeloneuropathy

G. Uziel, E. Bertini, P. Bardelli, M. Rimoldi, M. Gambetti

Research output: Contribution to journalArticlepeer-review


We report the biochemical and clinical results obtained during a dietary erucic acid (C22:1) therapy in 20 patients affected by X-linked adrenoleukodystrophy (ALD). Six patients were very severely affected, 9 had milder neurological symptoms and 5 were presymptomatic. Mean basal levels of plasma C26:0 were 1.41 ± 0.48 μg/ml in ALD patients (control values: 0.33 ± 0.12). In all patients C26:0 decreased to virtually normal values. In spite of good biochemical response and absence of consistent side effects of therapy, no encouraging data were observed during the clinical follow-up. The presymptomatic subjects were still free of symptoms after more than 1 year of therapy. The symptomatic patients, however worsened or did not show any improvement.

Original languageEnglish
Pages (from-to)274-279
Number of pages6
JournalDevelopmental Neuroscience
Issue number4-5
Publication statusPublished - 1991


  • Adrenoleukodystrophy
  • Adrenomyeloneuropathy
  • Erucic acid
  • Peroxisomal disorder
  • Very long chain fatty acids Dietary treatment

ASJC Scopus subject areas

  • Neuroscience(all)


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