High-dose enzyme replacement therapy attenuates cerebroventriculomegaly in a mouse model of mucopolysaccharidosis type II

Abstract

The natural progression of the severe form of mucopolysaccharidosis II in children is a rapid decline of neurodevelopmental function with hydrocephalus. Recombinant human iduronate-2-sulfatase enzyme replacement therapy (ERT) under a standard regimen seems to have limited effect. Therefore, we determined whether early, high-dose ERT attenuated… (More)
DOI: 10.1038/jhg.2013.92

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@article{Ahn2013HighdoseER, title={High-dose enzyme replacement therapy attenuates cerebroventriculomegaly in a mouse model of mucopolysaccharidosis type II}, author={So Yoon Ahn and Yun Sil Chang and Dong Kyung Sung and Ah-ra Ko and Chi Hwa Kim and Dong Kyeom Yoo and Keun Ho Lim and Young Bae Sohn and Dong Jin and Won Soon Park}, journal={Journal of Human Genetics}, year={2013}, volume={58}, pages={728-733} }