Clinical experience with miglustat therapy in pediatric patients with Niemann–Pick disease type C: A case series

Title
Clinical experience with miglustat therapy in pediatric patients with Niemann–Pick disease type C: A case series
Authors
Keywords
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Journal
MOLECULAR GENETICS AND METABOLISM
Volume 99, Issue 4, Pages 358-366
Publisher
Elsevier BV
Online
2009-11-30
DOI
10.1016/j.ymgme.2009.11.007

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