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Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year.

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Description scientific article published on 21 June 2007
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author: Ni-Chung Lee  Li-Kai Tsai  Yin-Hsiu Chien  Wuh-liang Hwu  Shyh-jye Chen 

Publication date June 21, 2007
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