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83 Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy 5 REFERENCES 1. Biffi A, Lucchini G, Rovelli A, Sessa M. Metachromatic leukodystrophy: an overview of current and prospective treatments. Bone Marrow Transplant. 2008;42:S2–S6. 2. van Rappard DF, Boelens JJ, Wolf NI. Metachromatic leukodystrophy: Disease spectrum and approaches for treatment. Best Pract Res Clin Endocrinol Metab. 2015;29:261–273. 3. Patil SA, Maegawa GH. Developing therapeutic approaches for metachromatic leukodystrophy. Drug Des Devel Ther. 2013;7:729–745. 4. Bayever E, Ladisch S, Philippart M et al. Bone-marrow transplantation for metachromatic leucodystrophy. Lancet. 1985;2:471–473. 5. KrivitW, ShapiroE, KennedyWet al.Treatment of late infantilemetachromatic leukodystrophy by bone marrow transplantation. N Engl J Med. 1990;322:28–32. 6. 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. 7. 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 Aug 7;10:94. 8. Batzios SP, Zafeiriou DI. Developing treatment options for metachromatic leukodystrophy. Mol Genet Metab. 2012;105:56–63. 9. Solders M, Martin DA, Andersson C et al. Hematopoietic SCT: a useful treatment for late metachromatic leukodystrophy. Bone Marrow Transplant. 2014;49(8):1046–1051 10. Bredius RG, Laan LA, Lankester AC et al. Early marrow transplantation in a pre-symptomatic neonatewith late infantilemetachromatic leukodystrophy does not halt disease progression. Bone Marrow Transplant. 2007;39:309–310. 11. Smith NJ, Marcus RE, Sahakian BJ, Kapur N, CoxTM. Haematopoietic stemcell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy. J Inherit Metab Dis. 2010;33:S471–475. 12. de Hosson LD, van deWarrenburg BP, Preijers FW et al. Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature. Bone Marrow Transplant. 2011;46:1071– 1076. 13. Boelens JJ, Orchard PJ, Wynn RF. Transplantation in inborn errors of metabolism: current considerations and future perspectives . Br J Haematol. 2014;167:293-303. 14. 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 15. Kehrer C, Blumenstock G, Raabe C, Krageloh-Mann I. Development and reliability of a classificationsystemforgrossmotorfunctioninchildrenwithmetachromaticleucodystrophy. Dev Med Child Neurol. 2011;53:156–160.