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

Chien, YHYHChienLee, NCNCLeeTsai, LKLKTsaiHuang, ACACHuangPeng, SFSFPengChen, SJSJChenHwu, WLWLHwu2011-02-142018-07-122011-02-142018-07-122007-06http://ntur.lib.ntu.edu.tw//handle/246246/224530en-USTreatment of Niemann-Pick disease type C in two children with miglustat: Initial responses and maintenance of effects over 1 year.