Adrenoleukodystrophy: effects of dietary restriction of very long chain fatty acids and of administration of carnitine and clofibrate on clinical status and plasma fatty acids

Brown, F.R.; Van Duyn, M.A.; Moser, A.B.; Schulman, J.D.; Rizzo, W.B.; Snyder, R.D.; Murphy, J.V.; Kamoshita, S.; Migeon, C.J.; Moser, H.W.

Johns Hopkins Medical Journal 151(4): 164-172

1982


ISSN/ISBN: 0021-7263
PMID: 7120720
Document Number: 192754
Six patients with biochemically confirmed adrenoleucodystrophy (ALD) and 1 man 36 years old with adrenomyeloneuropathy (AMN) took part in a clinical trial of dietary restriction of very long chain fatty acids for 4 months to 2 years. The diet did not decrease plasma C26:0 or C24:0 fatty acids in any of the patients and did not seem to change the clinical course of the disorder, except in the patient with AMN, who reported clinical improvement. Reasons for the negative results of the trial, at least in respect to childhood ALD, are discussed. It is concluded that endogenous sources of very long chain fatty acids are more important than exogenous sources in ALD.

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