A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy.
@article{Ahmed2016AMA, title={A model-based approach to assess the exposure-response relationship of Lorenzo's oil in adrenoleukodystrophy.}, author={M. Ahmed and R. Kartha and R. Brundage and J. Cloyd and C. Basu and B. Carlin and R. Jones and A. Moser and A. Fatemi and G. Raymond}, journal={British journal of clinical pharmacology}, year={2016}, volume={81 6}, pages={ 1058-66 } }
AIMS
X-linked adrenoleukodystrophy (X-ALD) is a peroxisomal disorder, most commonly affecting boys, associated with increased very long chain fatty acids (C26:0) in all tissues, causing cerebral demyelination and adrenocortical insufficiency. Certain monounsaturated long chain fatty acids including oleic and erucic acids, known as Lorenzo's oil (LO), lower plasma C26:0 levels. The aims of this study were to characterize the effect of LO administration on plasma C26:0 concentrations and to… CONTINUE READING
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References
SHOWING 1-10 OF 24 REFERENCES
Follow-up of 89 asymptomatic patients with adrenoleukodystrophy treated with Lorenzo's oil.
- Medicine
- Archives of neurology
- 2005
- 202
- PDF
Very long chain fatty acids in X‐linked adrenoleukodystrophy brain after treatment with Lorenzo's oil
- Biology, Medicine
- Annals of neurology
- 1994
- 70
Evaluation of the preventive effect of glyceryl trioleate-trierucate ("Lorenzo's oil") therapy in X-linked adrenoleukodystrophy: results of two concurrent trials.
- Medicine
- Advances in experimental medicine and biology
- 2003
- 47
A two-year trial of oleic and erucic acids ("Lorenzo's oil") as treatment for adrenomyeloneuropathy.
- Medicine
- The New England journal of medicine
- 1993
- 200
Elongation of very long-chain fatty acids is enhanced in X-linked adrenoleukodystrophy.
- Chemistry, Medicine
- Molecular genetics and metabolism
- 2005
- 76
X-linked adrenoleukodystrophy: clinical, metabolic, genetic and pathophysiological aspects.
- Biology, Medicine
- Biochimica et biophysica acta
- 2012
- 189
- PDF
Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls
- Biology, Medicine
- Annals of neurology
- 1999
- 254
Effect of erucic acid on platelets in patients with adrenoleukodystrophy.
- Biology, Medicine
- Biochemical and molecular medicine
- 1996
- 17