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171 Discussion, summary and future perspectives 10 REFERENCES 1. Hill DR, Bowery NG. 3H-baclofen and 3H-GABA bind to bicuculline-insensitive GABA B sites in rat brain. Nature . 1981;290:149-152. 2. Dralle D, Muller H, Zierski J, Klug N. Intrathecal baclofen for spasticity. Lancet. 1985;2:1003. 3. van Rappard DF, Boelens JJ, van Egmond ME, Kuball J, van Hasselt PM, Oostrom KJ, et al. Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience. Blood . 2016;127:3098-3101. 4. Groeschel S, Kuhl JS, Bley AE, Kehrer C, Weschke B, Doring M, et al. Long-term Outcome of AllogeneicHematopoietic StemCellTransplantation in PatientsWith JuvenileMetachromatic Leukodystrophy Compared With Nontransplanted Control Patients. JAMA Neurol . 2016. doi:10.1001/jamaneurol.2016.2067 5. Boucher AA, Miller W, Shanley R, Ziegler R, Lund T, Raymond G, 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;10:94-0313. 6. Baird K, Cooke K, Schultz KR. Chronic graft-versus-host disease (GVHD) in children. Pediatr Clin North Am . 2010;57:297-322. 7. van Rappard DF, Klauser A, Steenweg ME, Boelens JJ, Bugiani M, van der Knaap MS, et al. Quantitative MR spectroscopic imaging in metachromatic leukodystrophy: value for prognosis and treatment. J Neurol Neurosurg Psychiatry . 2017. doi: 10.1136/jnnp-2017- 316364. 8. MartinHR,PoeMD,ProvenzaleJM,KurtzbergJ,MendizabalA,EscolarML.Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy. Biol Blood Marrow Transplant. 2013;19:616-624. 9. Kim J, Sun Z, Ezekian B, Schooler GR, Prasad VK, Kurtzberg J, et al. Gallbladder abnormalities in children with metachromatic leukodystrophy. J Surg Res . 2017;208:187-1991. 10. Matzner U, Hartmann D, Lullmann-Rauch R, Coenen R, Rothert F, Mansson JE, et al. Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations. Gene Ther. 2002;9:53-63. 11. Dali CI, Barton NW, FarahMH, MoldovanM, Mansson JE, Nair N, et al. Sulfatide levels correlate with severity of neuropathy in metachromatic leukodystrophy. Ann Clin Transl Neurol. 2015;2:518-533. 12. Krageloh-Mann I, Groeschel S, Kehrer C, Opherk K, Nagele T, Handgretinger R, et al. Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort. Bone Marrow Transplant. 2013;48:369-375. 13. van Rappard DF, Bugiani M, Boelens JJ, van der Steeg AF, Daams F, de Meij TG, et al. Gallbladder and the risk of polyps and carcinoma in metachromatic leukodystrophy . Neurology . 2016;87:103-111.