Mol Genet Metab. 2010 May;100(1):20-3. Epub 2010 Jan 4.

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

Valayannopoulos V, Boddaert N, Barbier V, Le Merrer M, Caillaud C, de Lonlay P.

Source

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; [PubMed - indexed for MEDLINE]

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Iduronidase

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Anthropometric data of 14 patients with mucopolysaccharidosis I: retrospective analysis and efficacy of recombinant human alpha-L-iduronidase (laronidase). [Mol Genet Metab. 2010]
Anthropometric data of 14 patients with mucopolysaccharidosis I: retrospective analysis and efficacy of recombinant human alpha-L-iduronidase (laronidase).
Tylki-Szymanska A, Rozdzynska A, Jurecka A, Marucha J, Czartoryska B. Mol Genet Metab. 2010 Jan; 99(1):10-7.
Outcome after three years of laronidase enzyme replacement therapy in a patient with Hurler syndrome. [J Inherit Metab Dis. 2006]
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Thomas JA, Jacobs S, Kierstein J, Van Hove J. J Inherit Metab Dis. 2006 Dec; 29(6):762. Epub 2006 Nov 6.
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Review Laronidase treatment of mucopolysaccharidosis I.
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Review Laronidase. [BioDrugs. 2002]
Review Laronidase.
. BioDrugs. 2002; 16(4):316-8.

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