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Bezafibrate for X-linked adrenoleukodystrophy.
Engelen M, Tran L, Ofman R, Brennecke J, Moser AB, Dijkstra IM, Wanders RJ, Poll-The BT, Kemp S. Engelen M, et al. PLoS One. 2012;7(7):e41013. doi: 10.1371/journal.pone.0041013. Epub 2012 Jul 20. PLoS One. 2012. PMID: 22911730 Free PMC article. Clinical Trial.
Disease progression in women with X-linked adrenoleukodystrophy is slow.
Huffnagel IC, Dijkgraaf MGW, Janssens GE, van Weeghel M, van Geel BM, Poll-The BT, Kemp S, Engelen M. Huffnagel IC, et al. Orphanet J Rare Dis. 2019 Feb 7;14(1):30. doi: 10.1186/s13023-019-1008-6. Orphanet J Rare Dis. 2019. PMID: 30732635 Free PMC article.
X-linked adrenoleukodystrophy in women: a cross-sectional cohort study.
Engelen M, Barbier M, Dijkstra IM, Schür R, de Bie RM, Verhamme C, Dijkgraaf MG, Aubourg PA, Wanders RJ, van Geel BM, de Visser M, Poll-The BT, Kemp S. Engelen M, et al. Brain. 2014 Mar;137(Pt 3):693-706. doi: 10.1093/brain/awt361. Epub 2014 Jan 29. Brain. 2014. PMID: 24480483
Reply: Age-dependent penetrance among females with X-linked adrenoleukodystrophy.
Engelen M, Barbier M, Dijkstra IM, Schür R, de Bie RM, Verhamme C, Dijkgraaf MG, Aubourg PA, Wanders RJ, van Geel BM, de Visser M, Poll-The BT, Kemp S. Engelen M, et al. Brain. 2015 Feb;138(Pt 2):e326. doi: 10.1093/brain/awu233. Epub 2014 Aug 22. Brain. 2015. PMID: 25149411 No abstract available.
Cholic acid therapy in Zellweger spectrum disorders.
Berendse K, Klouwer FC, Koot BG, Kemper EM, Ferdinandusse S, Koelfat KV, Lenicek M, Schaap FG, Waterham HR, Vaz FM, Engelen M, Jansen PL, Wanders RJ, Poll-The BT. Berendse K, et al. J Inherit Metab Dis. 2016 Nov;39(6):859-868. doi: 10.1007/s10545-016-9962-9. Epub 2016 Jul 28. J Inherit Metab Dis. 2016. PMID: 27469511 Free PMC article.
252 results