Treatment of Niemann-Pick disease type C in two children with miglustat: Initial responses and maintenance of effects over 1 year.
Resource
J Inherit Metab Dis,63,1-8.
Journal
J Inherit Metab Dis
Journal Issue
63
Pages
1-8
Date Issued
2007-06
Date
2007-06
Author(s)
Chien, YH
Lee, NC
Tsai, LK
Huang, AC
Peng, SF
Chen, SJ
Hwu, WL