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Mol Genet Metab. 2010 May;100(1):20-3. doi: 10.1016/j.ymgme.2009.12.019. Epub 2010 Jan 4.

Cognitive and neuroradiological improvement in three patients with attenuated MPS I treated by laronidase.

Author information

1
Reference Center for Inherited Metabolic Disorders, Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades Hospital, University Paris Descartes, Paris, France. vassili.valaya@nck.aphp.fr

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.

PMID:
20106688
DOI:
10.1016/j.ymgme.2009.12.019
[Indexed for MEDLINE]

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