14800-DvRappard

119 Quantitative MR spectroscopic imaging in metachromatic leukodystrophy 7 REFERENCES 1. van Rappard DF, Boelens JJ, Wolf NI. Metachromatic leukodystrophy: Disease spectrum and approaches for treatment. Best Pract Res Clin Endocrinol Metab 2015;29:261-73. 2. Eckhardt M. The role and metabolism of sulfatide in the nervous system. Mol Neurobiol 2008;37:93-103. 3. Biffi A, Lucchini G, Rovelli A, Sessa M. Metachromatic leukodystrophy: an overview of current and prospective treatments. Bone Marrow Transplant 2008;42 (Suppl 2):S2-S6. 4. Boucher AA, Miller W, Shanley R, et al . Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report. Orphanet J Rare Dis 2015;10:94. 5. Biffi A, Montini E, Lorioli L, et al . Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 2013;341:1233158. 6. Eichler F, Grodd W, Grant E, et al. Metachromatic leukodystrophy: a scoring system for brain MR imaging observations. Am J Neuroradiol 2009;30:1893-1897. 7. van Rappard DF, Boelens JJ, van Egmond ME, et al . Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience. Blood 2016;127:3098-3101. 8. Groeschel S, Kuhl JS, Bley AE, et al . Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Juvenile Metachromatic Leukodystrophy Compared With Nontransplanted Control Patients. JAMA Neurol 2016;73:1133-1140. 9. Oz G, Alger JR, Barker PB, et al . Clinical proton MR spectroscopy in central nervous system disorders. Radiology 2014;270:658-679. 10. van der Voorn JP, Pouwels PJ, Hart AA, et al . Childhood white matter disorders: quantitative MR imaging and spectroscopy. Radiology 2006;241:510-517. 11. Kruse B, Hanefeld F, Christen HJ, et al . Alterations of brain metabolites in metachromatic leukodystrophy as detected by localized proton magnetic resonance spectroscopy in vivo. J Neurol 1993;241:68-74. 12. van Egmond ME, Pouwels PJ, Boelens JJ, et al . Improvement of white matter changes on neuroimaging modalities after stem cell transplant in metachromatic leukodystrophy. JAMA Neurol 2013;70:779-782. 13. Kehrer C, Blumenstock G, Raabe C, Krageloh-Mann I. Development and reliability of a classificationsystemforgrossmotorfunctioninchildrenwithmetachromaticleucodystrophy. Dev Med Child Neurol 2011;53:156-160. 14. Steenweg ME, Wolf NI, van Wieringen WN, et al . Quantitative MRI in hypomyelinating disorders: Correlation with motor handicap. Neurology 2016;87:752-758. 15. Natt O, Bezkorovaynyy V, Michaelis T, Frahm J. Use of phased array coils for a determination of absolute metabolite concentrations. Magn Reson Med 2005;53:3-8.

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