14800-DvRappard

71 Intrathecal baclofen treatment: metachromatic leukodystrophy versus spastic cerebral palsy 4 REFERENCES 1. Biffi A, Lucchini G, Rovelli A, Sessa M. Metachromatic leukodystrophy: an overview of current and prospective treatments. Bone Marrow Transplant. 2008;42 Suppl 2:S2-6. 2. Eckhardt M. The role and metabolism of sulfatide in the nervous system. Mol Neurobiol. 2008;37:93-103. 3. Patil SA, Maegawa GH. Developing therapeutic approaches for metachromatic leukodystrophy. Drug Des Devel Ther . 2013;7:729-745. 4. 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. 5. Groeschel S, Kuhl JS, Bley AE, Kehrer C, Weschke B, Doring M, 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. 6. 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. 7. Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, et al. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet. 2016;388;476-487. 8. Kehrer C, Blumenstock G, Raabe C, Krageloh-Mann I. Development and reliability of a classificationsystemforgrossmotorfunctioninchildrenwithmetachromaticleucodystrophy. Dev Med Child Neurol . 2011;53:156-60. 9. Palisano R, Rosenbaum P, Walter S, Russell D, Wood E, Galuppi B. Development and reliability of a system to classify gross motor function in children with cerebral palsy. Dev Med Child Neurol . 1997;39(4):214-23. 10. Jacobs JM. Management options for the child with spastic cerebral palsy. Orthop Nurs . 2001;20:53-9; quiz 9-61. 11. Tilton A. Management of spasticity in children with cerebral palsy. Semin Pediatr Neurol. 2009;16:82-9. 12. Hill DR, Bowery NG. 3H-baclofen and 3H-GABA bind to bicuculline-insensitive GABA B sites in rat brain. Nature. 1981;290:149-152. 13. Albright AL, Barry MJ, Shafton DH, Ferson SS. Intrathecal baclofen for generalized dystonia . Dev Med Child Neurol . 2001;43(10):652-7. 14. Dralle D, Muller H, Zierski J, Klug N. Intrathecal baclofen for spasticity. Lancet. 1985;2:1003. 15. Albright AL, Gilmartin R, Swift D, Krach LE, Ivanhoe CB, McLaughlin JF. Long-term intrathecal baclofen therapy for severe spasticity of cerebral origin. J Neurosurg. 2003;98:291-5.

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