Favorable outcomes following early onset oral miglustat in early infantile Niemann Pick Type C
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference17 articles.
1. Niemann-Pick disease Type C;Vanier;Orphanet. J. Rare. Dis.,2010
2. Niemann-Pick Disease Type C;Patterson,2000
3. Recommendations for the diagnosis and management of Niemann-Pick disease Type C: an update;Patterson;Mol. Genet. Metab.,2012
4. Recommendations on the diagnosis and management of Niemann-Pick disease Type C;Wraith;Mol. Genet. Metab.,2009
5. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study;Patterson;Lancet Neurol.,2007
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5. Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study;Orphanet Journal of Rare Diseases;2023-07-21
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