Bezafibrate for X-linked adrenoleukodystrophy

PLoS ONE

Volumn 7, Issue 7, 2012, Pages

  Engelen, Marc ^a   Tran, Luc ^a   Ofman, Rob ^a   Brennecke, Josephine ^a   Moser, Ann B ^b   Dijkstra, Inge M E ^a   Wanders, Ronald J A ^a   Poll The, Bwee Tien ^a   Kemp, Stephan ^a  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

^b KENNEDY KRIEGER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BEZAFIBRATE; HIGH DENSITY LIPOPROTEIN; LOW DENSITY LIPOPROTEIN; TRIACYLGLYCEROL; VERY LONG CHAIN FATTY ACID;

ADRENOLEUKODYSTROPHY; ARTICLE; BLOOD SAMPLING; CELL LEVEL; CLINICAL ARTICLE; CLINICAL EFFECTIVENESS; CLINICAL TRIAL; DRUG BLOOD LEVEL; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; FATTY ACID BLOOD LEVEL; HUMAN; HUMAN CELL; LIPID STORAGE; LYMPHOCYTE; MALE; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; TREATMENT DURATION; TRIACYLGLYCEROL BLOOD LEVEL; UNSPECIFIED SIDE EFFECT;

ADRENOLEUKODYSTROPHY; BEZAFIBRATE; HUMANS; HYPOLIPIDEMIC AGENTS; MALE; TREATMENT OUTCOME;

EID: 84864113240     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0041013     Document Type: Article

References (10)

1. Moser HW, Mahmood A, Raymond GV, (2007) X-linked adrenoleukodystrophy. Nat Clin Pract Neurol 3: 140-151.
2. Mosser J, Douar AM, Sarde CO, Kioschis P, Feil R, et al. (1993) Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters. Nature 361: 726-730.
3. Ofman R, Dijkstra IM, van Roermund CW, Burger N, Turkenburg M, et al. (2010) The role of ELOVL1 in very long-chain fatty acid homeostasis and X-linked adrenoleukodystrophy. EMBO Mol Med 2: 90-97.
4. Engelen M, Schackmann MJ, Ofman R, Sanders RJ, Dijkstra IM, et al. (2012) Bezafibrate lowers very long-chain fatty acids in X-linked adrenoleukodystrophy fibroblasts by inhibiting fatty acid elongation. J Inherit Metab Dis. 10.1007/s10545-012-9471-4 [doi].
5. Miller DB, Spence JD, (1998) Clinical pharmacokinetics of fibric acid derivatives (fibrates). Clin Pharmacokinet 34: 155-162.
6. Valianpour F, Selhorst JJ, van Lint LE, van Gennip AH, Wanders RJ, et al. (2003) Analysis of very long-chain fatty acids using electrospray ionization mass spectrometry. Mol Genet Metab 79: 189-196.
7. Masnatta LD, Cuniberti LA, Rey RH, Werba JP, (1996) Determination of bezafibrate, ciprofibrate and fenofibric acid in human plasma by high-performance liquid chromatography. J Chromatogr B Biomed Appl 687: 437-442.
8. Hubbard WC, Moser AB, Liu AC, Jones RO, Steinberg SJ, et al. (2009) Newborn screening for X-linked adrenoleukodystrophy (X-ALD): validation of a combined liquid chromatography-tandem mass spectrometric (LC-MS/MS) method. Mol Genet Metab 97: 212-220.
9. Kemp S, Berger J, Aubourg P, (2012) X-linked adrenoleukodystrophy: Clinical, metabolic, genetic and pathophysiological aspects. Biochim Biophys Acta. 10.1016/j.bbadis.2012.03.012 [doi].
10. Engelen M, Ofman R, Dijkgraaf MGW, Hijzen M, van der Wardt LA, et al. (2010) Lovastatin in X-Linked Adrenoleukodystrophy. N Engl J Med 362: 276-277.