| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10834070 | Molecular Genetics and Metabolism | 2010 | 4 Pages |
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 2Â years; Patients 1 and 3 displayed improvement on MRI scans of the brain.
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Authors
V. Valayannopoulos, N. Boddaert, V. Barbier, M. Le Merrer, C. Caillaud, P. de Lonlay,