Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome
Author:
Funder
National Institutes of Health
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Genetics(clinical),General Medicine
Link
http://link.springer.com/content/pdf/10.1186/s13023-018-0817-3.pdf
Reference29 articles.
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2. Aldenhoven M, Wynn RF, Orchard PJ, O’Meara A, Veys P, Fischer A, Valayannopoulos V, Neven B, Rovelli A, Prasad VK. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study. Blood. 2015;125:2164–72.
3. Poe MD, Chagnon SL, Escolar ML. Early treatment is associated with improved cognition in Hurler syndrome. Ann Neurol. 2014;76:747–53.
4. Kunin-Batson A, Shapiro E, Rudser K, Lavery C, Bjoraker K, Jones S, Wynn R, Vellodi A, Tolar J, Orchard P. Long-term cognitive and functional outcomes in children with Mucopolysaccharidosis (MPS)-IH (Hurler syndrome) treated with hematopoietic cell transplantation J Inherit Metab Dis Rep. 2016;29:1–8.
5. Clarke LA, Atherton AM, Burton BK, Day-Salvatore DL, Kaplan P, Leslie ND, Scott CR, Stockton DW, Thomas JA, Muenzer J. Mucopolysaccharidosis type I newborn screening: best practices for diagnosis and management. J Pediatr. 2017;182:363–70.
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