| Literature DB >> 20106688 |
V Valayannopoulos1, N Boddaert, V Barbier, M Le Merrer, C Caillaud, P de Lonlay.
Abstract
Stem cell transplantation is not appropriate first-line treatment for attenuated phenotypes of mucopolysaccharidosis type I (MPS I). In three patients with attenuated MPSA I treated by laronidase, Patients 2 and 3 displayed significant cognitive improvement within 2years; Patients 1 and 3 displayed improvement on MRI scans of the brain. (c) 2010. Published by Elsevier Inc.Entities:
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Year: 2010 PMID： 20106688 DOI： 10.1016/j.ymgme.2009.12.019
Source DB: PubMed Journal: Mol Genet Metab ISSN： 1096-7192 Impact factor: 4.797