Document Type

Journal Article

Publication Date

12-1-2015

Journal

Molecular Genetics and Metabolism Reports

Volume

5

Inclusive Pages

103-106

DOI

10.1016/j.ymgmr.2015.11.001

Abstract

In mucopolysaccharidoses (MPS), glycosaminoglycans (GAG) accumulate in tissues. In MPS II, approximately two-thirds of patients are cognitively impaired. We investigated levels of GAG in cerebrospinal fluid (CSF) in different populations from four clinical studies (including NCT00920647 and NCT01449240). Data indicate that MPS II patients with cognitive impairment have elevated levels of CSF GAG, whereas those with the attenuated phenotype typically have levels falling between those of the cognitively affected patients and healthy controls.

Comments

Reproduced with permission of Elsevier B.V. Molecular Genetics and Metabolism Reports

Creative Commons License

Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 License.

Peer Reviewed

1

Open Access

1

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